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301 Moved Permanently - mda75.com
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Date: Fri, 23 May 2025 21:18:39 GMT
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HTTP Kodu 301 Kalıcı Olarak Taşındı
301 durum kodu, istenen kaynağın kalıcı olarak yeni bir URL'ye taşındığı anlamına gelir. Gelecekteki tüm istekler yeni adresi kullanmalıdır.
Kod 301 ne zaman kullanılır?
- Bir web sitesi alan adını değiştirirken
- URL yapılarını değiştirirken
- SEO için yönlendirmeleri ayarlarken
Kod 301 kullanıcı için ne anlama geliyor?
Tarayıcı kullanıcıyı otomatik olarak yeni adrese yönlendirecek ve arama motorları dizinlerini güncelleyecektir.
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<meta name="keywords" content="MDA 75th anniversary, muscular dystrophy, advocacy, community support, ALS, gene therapy, research, donate">
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<title>Join MDA's 75th Anniversary: Advocate, Support, and Transform Lives</title>
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<h1>75 YEARS OF <br><span>Legacy. Impact. <br />Momentum.</span></h1>
<p>For 75 years, MDA’s legacy has been defined by an extraordinary community committed to progress. Together, we’ve transformed research, championed legislation, and supported access to care and resources for people living with neuromuscular diseases. This year, we celebrate their dedication and all we’ve achieved together.</p>
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<h3>The legacy of <br />a community committed</h3>
<p>Research. Policy. Opportunity. Freedom. As we celebrate 75 years of progress, we have no intention of slowing down. We’re building on this momentum by:</p>
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<li>Expanding research into gene therapies and muscle regeneration</li>
<li>Advocating for policies that empower people with neuromuscular disease</li>
<li>Supporting access to medical care through MDA’s Care Center Network</li>
<li>Continuing to build and empower a strong, connected community</li>
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<p><b>Scroll down and swipe left or right</b> to explore the moments in which we’ve made history—and the impacts that have been felt by millions.</p>
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<h2>1949</h2>
<h3>Dr. Ade Milhorat is one of few doctors researching muscular dystrophies worldwide</h3>
<p>In 1949, Dr. Ade Milhorat of New York Hospital-Cornell Medical Center was one of only a handful of doctors in the world researching muscular dystrophies. His research work lost funding, and it might well have been the end of his efforts were it not for a friend who also had muscular dystrophy. That friend was Paul Cohen. </p>
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<h2>1950</h2>
<h3>The Muscular Dystrophy Association of America is founded</h3>
<p>On June 6, 1950, Paul Cohen, a prominent New York businessman with a form of muscular dystrophy (FSH), invited a group of individuals to meet in his Rye, NY office. Each had a personal connection to muscular dystrophy, and the gathering focused on the urgent need to raise funds to advance research seeking treatments and cures for muscular dystrophy. The group formed the Muscular Dystrophy Association of America (MDAA)*, and two months later, MDA made its first research grant to support Dr. Milhorat’s research program.</p>
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<h2>1952</h2>
<h3>Dean Martin, Jerry Lewis promote MDA on national radio</h3>
<p>During their January 4, 1952 radio program, Martin and Lewis make an appeal for nationwide support of MDA. Later that year, Martin and Lewis were named MDA National Co-Chairmen.</p>
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<h2>1952</h2>
<h3>MDA Ambassador Program launched</h3>
<p>MDA introduces its first Ambassador Program to raise awareness about muscular dystrophy and other neuromuscular disorders. Over time, the program evolves to highlight the strength and diverse stories of people living with neuromuscular disease in the MDA community.</p>
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<h2>1952</h2>
<h3>Five MDA Local Telethons Held in Two Years</h3>
<p>Early to recognize the power of television to bolster awareness and raise income for the Association, MDA’s top fundraising priority was the pioneering work of establishing local Telethons featuring a variety of celebrities. Thanks to the help of top stars like Robert Alda, Dick Van Dyke, Captain Video and Virginia Graham, MDA successfully hosted five local Telethons in two years in Cleveland; Washington DC; Atlanta; Grand Rapids; and Madison, Wisconsin.</p>
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<h2>1952</h2>
<h3>The International Association Resolves to Fight Muscular Dystrophy and "Fills the Boot"</h3>
<p>Early in 1952, a group of families affected by muscular dystrophy approached Local 718 fire station in the Boston area to ask professional fire fighters to help fight muscular dystrophy. Responding enthusiastically, the fire fighters took to the streets with their boots in hand to ask greater Boston to make donations that would be used to fight muscular dystrophy. The Fill the Boot campaign was an instant success and on August 19, 1954, the International Association of Fire Fighters (IAFF) membership passed a resolution to support the Association’s fight against muscular dystrophy until treatments and cures are found.</p>
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<h2>1953</h2>
<h3>The first MDA Care Centers are established</h3>
<p>The first MDA Care Centers are established at NYU Langone Health and University of Rochester Medical Center, providing specialized neuromuscular disease support and care—a foundation of MDA’s Care Center Network.</p>
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<h2>1953</h2>
<h3>National Association of Letter Carriers becomes MDA's first national sponsor</h3>
<p>MDA Founder Paul Cohen recruits the National Association of Letter Carriers (NALC) as the organization’s first national sponsor. The NALC quickly established a nationwide door-to-door campaign for MDA, the first of which took place on November 25, 1953. The inaugural campaign was heralded by a special two-hour coast to coast television show hosted by Martin and Lewis.</p>
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<h2>1955</h2>
<h3>Eleanor Gehrig named MDA's National Campaign Chairperson
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<p>After losing her husband, New York Yankees icon Lou Gehrig, to ALS, Eleanor Gehrig made it her mission to help others with the disease. To do so, she partnered with MDA, spearheading advocacy and care efforts that changed the future of life with ALS for good.</p>
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<h2>1955</h2>
<h3>MDA holds first MDA Summer Camp</h3>
<p>MDA holds its first Summer Camp with 16 campers in Sussex, NJ, creating a unique place for children with neuromuscular diseases to experience independence, build friendships, and have fun in an accessible environment.</p>
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<h2>1956</h2>
<h3>Martin and Lewis Local Telethon for MDA at Carnegie Hall</h3>
<p>Martin and Lewis co-hosted the 1956 Telethon at New York City’s famed Carnegie Hall and broadcast June 29-30 on Dumont Station WABD (now WABC) in New York, raising some $600,000. </p>
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<h2>1966</h2>
<h3>MDA's Legendary Labor Day Telethon Series Begins</h3>
<p> The first MDA Jerry Lewis Labor Day Telethon was broadcast in 1966 by a single station, WNEW-TV in New York. The telecast, which resulted in $1,002,114 being pledged, was so successful that MDA selected Labor Day weekend for its future Telethons.</p>
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<h2>1971</h2>
<h3>MDA Labor Day Telethon seen across the country</h3>
<p>Robert M. Bennett (President of Metromedia Broadcasting), creates the “Love Network” of stations that facilitates the nation’s first networked Telethon in 1971.</p>
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<h2>1983</h2>
<h3>The Orphan Drug Act (ODA) of 1983 enacted.</h3>
<p>The Orphan Drug Act (ODA) of 1983 was enacted to provide incentives to support research and development into drugs for rare diseases with small patient populations.</p>
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<h2>1986</h2>
<h3>CITGO Joins Forces with MDA</h3>
<p>CITGO Petroleum Corporation begins its enduring partnership with the Muscular Dystrophy Association. Over the decades, CITGO employees, marketers, and retailers have supported MDA through volunteer efforts and community engagement, helping to advance critical programs such as MDA Care Centers, summer camps, and research initiatives.</p>
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<h2>1986</h2>
<h3>MDA-funded discovery of the DMD gene launches a new era in genetic research and therapies.</h3>
<p>In 1986, MDA-funded researcher Dr. Lou Kunkel and his team identified the gene responsible for Duchenne and Becker muscular dystrophy, a landmark discovery that paved the way for targeted research and innovative treatments. This breakthrough marked the beginning of the gene discovery era, transforming scientific understanding of neuromuscular diseases and laying the foundation for advancements in genetic therapies that continue to benefit the neuromuscular community today.</p>
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<h2>1988</h2>
<h3>MDA funds SMA Cell Bank to advance gene discovery</h3>
<p>MDA funds Dr. Conrad Gilliam and collaborators to establish the SMA Cell Bank, accelerating the discovery of the SMA-causing gene through groundbreaking natural history studies.</p>
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<h2>1990</h2>
<h3>Americans with Disabilities Act (ADA) signed into law</h3>
<p>On July 26, 1990, President George H.W. Bush signed into law this landmark civil rights legislation intended to eliminate barriers for people with disabilities. Driven by the advocacy work of MDA and other organizations, this pivotal law represents a critical milestone in advancing the rights and independence of people living with neuromuscular diseases.</p>
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<h2>1992</h2>
<h3>Genetic mutations causing myotonic dystrophy type 1 identified</h3>
<p>In 1992, MDA supported research leading to the identification of genetic mutations causing myotonic dystrophy type 1 (DM1)</p>
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<h2>1993</h2>
<h3>Mutations in PMP22 identified as a causative for CMT1A</h3>
<p>In 1993, mutations in PMP22 were identified as a causative for CMT1A (first localized to 17p11.2) by a collaborative group sponsored by MDA.</p>
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<h2>1995</h2>
<h3>FDA approves Riluzole, first treatment for ALS</h3>
<p>In 1995, the FDA approved riluzole, the first-ever treatment for ALS, offering new hope for people living with the disease. Backed by years of research, including contributions from MDA-supported scientists, riluzole’s approval represented a critical step in ALS treatment, paving the way for further advancements in therapies aimed at improving quality of life and extending survival for people living with ALS.</p>
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<h2>1996</h2>
<h3>American Medical Association honors MDA with the Lifetime Achievement Award</h3>
<p>In 1996, the American Medical Association honored MDA with the prestigious Lifetime Achievement Award, recognizing decades of pioneering work in neuromuscular disease research, care, and advocacy. This accolade underscores MDA's enduring commitment to leading efforts that have shaped the landscape of neuromuscular medicine.</p>
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<h2>1998</h2>
<h3>MDA-funded work leads to three FDA-approved exon skipping drugs for Duchenne muscular dystrophy</h3>
<p>In 1998. MDA-funded research contributed to the development of three FDA-approved exon-skipping drugs for Duchenne muscular dystrophy, offering targeted treatment options for specific genetic mutations within the disorder. These therapies marked a significant advancement in Duchenne care, slowing disease progression for eligible individuals and opening doors to further genetic-based approaches.</p>
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<h2>1999</h2>
<h3>MDA funds first clinical trial for gene therapy for any type of muscular dystrophy.</h3>
<p>In 1999, the Muscular Dystrophy Association funded the first clinical trial for gene therapy for any type of muscular dystrophy. This trial was led by Dr. Jerry Mendell and was the first gene therapy trial for muscular dystrophy.</p>
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<h2>2000</h2>
<h3>MDA funds research to boost SMN2 gene expression for SMA treatment.</h3>
<p>In 2000, MDA funded Dr. Christian Lorson (Arizona State University) to investigate factors that can help increase expression of full length SMN protein from the SMN2 gene. </p>
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<h2>2001</h2>
<h3>Genetic mutation causing myotonic dystrophy type 2 identified.</h3>
<p>In 2001, a team led by Dr. Thomas R. Ranum and Dr. Maurice Day discovered the CCTG tetranucleotide repeat expansion in the CNBP (ZNF9) gene responsible for DM2. </p>
<a href="https://www.mda.org/science/research-grants" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2001</h2>
<h3>U.S. Congress Passes the MD-CARE Act</h3>
<p>The 2001 passage of the MD CARE Act, championed by MDA advocates and allies, marked a pivotal achievement in the fight against muscular dystrophy by expanding federal support for research, care, and resources across the country. </p>
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<h2>2003</h2>
<h3>The Centers of Excellence program in muscular dystrophy research is established by NIH</h3>
<p>The Centers of Excellence program in muscular dystrophy research was established in 2003 by NIH in honor of the late Senator Paul D. Wellstone of Minnesota. </p>
<p>The Wellstone Centers focus on translating basic research into clinical applications, enhancing diagnostics, and developing new therapies, with an emphasis on collaboration across institutions. </p>
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<h2>2006</h2>
<h3>MDA funds the first-ever gene therapy trial for Duchenne muscular dystrophy</h3>
<p>In 2006, MDA funded the first-ever gene therapy trial for Duchenne muscular dystrophy, marking a transformative moment in the pursuit of neuromuscular disease treatments. This pioneering trial paveed the way for gene-based approaches aimed at addressing the root causes of Duchenne, opening the door to innovative therapies for a range of neuromuscular diseases. </p>
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<h2>2006</h2>
<h3>FDA approves Lumizyme (alglucosidase alfa) for the treatment of Pompe Disease</h3>
<p>In 2006, the FDA approved Lumizyme, a groundbreaking enzyme replacement therapy for Pompe disease. The development of the therapy, funded in part by MDA, marked a significant advancement in care, offering new hope for improved quality of life.</p>
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<h2>2007</h2>
<h3>MDA funds Dr. Adrian Krainer's antisense therapy research, leading to Spinraza for SMA</h3>
<p>In 2007, MDA funded pioneering research by Dr. Adrian Krainer in antisense therapy, leading to a breakthrough in treatment for spinal muscular atrophy (SMA). Dr. Krainer’s work culminates in the development and FDA approval of Spinraza, the first therapy to effectively target the genetic root of SMA, transforming SMA from a terminal diagnosis in young children to a manageable condition.</p>
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<h2>2010</h2>
<h3>Surveying New Therapeutive Avenues</h3>
<p>In 2010, MDA awarded multiple grants to explore SMA gene therapy, gene delivery, novel ASO therapies, and new small molecule targets in mouse and zebrafish models of SMA.</p>
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<h2>2011</h2>
<h3>Identification of C9ORF mutations in ALS</h3>
<p>In 2011, MDA-funded research led to the identification of C9ORF72 gene mutation as the most common genetic cause of ALS and FTD. </p>
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<h2>2015</h2>
<h3>MDA makes the difficult decision to end its historic Telethon tradition</h3>
<p>As families and supporters began looking for new ways to support and get involved with the organization, MDA embraced new opportunities through social media and other digital channels to inspire the nation in support of the fight against neuromuscular diseases.</p>
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<h2>2015</h2>
<h3>FDA approves Keveyis for treating periodic paralysis</h3>
<p>In 2015, the FDA approved MDA-funded Keveyis (Dichlorphena-mide) for the treatment of hyperkalemic and hypokalemic periodic paralysis.</p>
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<h2>2015</h2>
<h3>MDA establishes the MDA Resource Center to provide trusted guidance and support nationwide</h3>
<p>In 2015, MDA established the MDA Resource Center as the organization’s national information hub, offering individuals and families a reliable source of information, guidance, and support to navigate life with neuromuscular disease. </p>
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<h2>2016</h2>
<h3>FDA approves Exondys 51 (eteplirsen) for the treatment of DMD</h3>
<p>After decades of funding Duchenne muscular dystrophy research, the MDA-supported landmark treatment, Exondys 51 (eteplirsen), was approved by the FDA in 2016. This therapy was the first approved drug to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. </p>
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<h2>2016</h2>
<h3>FDA approves Spinraza (nusinersen) for the treatment of SMA</h3>
<p>In 2016, the FDA issued a landmark approval for Spinraza, providing life-changing therapeutic options for indivisuals with SMA. With foundational support from MDA, this treatment offers the potential to slow disease progression and improve motor function. </p>
<a href="https://mdaquest.org/a-revolutionary-decade-for-spinal-muscular-atrophy-therapies/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2017</h2>
<h3>FDA approves Emflaza (deflazacort) for the treatment of Duchenne Muscular Dystrophy</h3>
<p>In 2017, the FDA approved Emflaza, a signature treatment for the most common form of childhood muscular dystrophy, Duchenne, after decades of MDA research, funding, and trials. </p>
<a href="https://www.mda.org/press-releases/mda-celebrates-news-fda-approval-emflaza-treatment-duchenne-muscular-dystrophy" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2017</h2>
<h3>FDA approves Radicava (edaravone) for the treatment of ALS</h3>
<p>After completing human trials at more than a dozen MDA care centers, the FDA approved Radicava for the treatment of ALS in 2017. </p>
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<h2>2017</h2>
<h3>FDA approves Soliris (eculizumab) for the treatment of myasthenia gravis</h3>
<p>In 2017, the FDA approved Soliris for the treatment of myasthenia gravis. A first-of-its-kind complement inhibitor drug, this therapeutic strategy has paved the way for new care options for the MDA community, and new paths for MDA-funded research. </p>
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<h2>2018</h2>
<h3>Decoding Disease Mechanisms</h3>
<p>In 2018, MDA funded grants to Dr. Arthur Burghes (Ohio State) for identifying modifiers of disease and Dr. Rashmi Kothary(Ottawa Hospital Research Institute) to study abnormal metabolism in SMA. </p>
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<h2>2018</h2>
<h3>MDA launches the MOVR Data Hub™</h3>
<p>In 2018, MDA launched the MOVR (NeuroMuscular ObserVational Research) Data Hub™, a neuromuscular disease data platform that centralizes clinical information from MDA Care Centers across the country. This comprehensive resource empowers researchers, clinicians, and healthcare providers with real-time insights, accelerating progress in understanding disease patterns, treatment outcomes, and patient needs. </p>
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<h2>2018</h2>
<h3>Adrian Krainer, PhD, wins Breakthrough Prize for developing Spinraza, an SMA therapy</h3>
<p>In 2018, MDA researcher Adrian Krainer, PhD, received the Breakthrough Prize in Life Sciences for the development of an effective antisense oligonucleotide therapy (Spinraza) for children with SMA.Dr. Krainer’s work with Spinraza underscores the power of targeted genetic therapies and sets a precedent for future innovations in treating neuromuscular diseases.</p>
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<h2>2018/2019</h2>
<h3>FDA approves Firdapse/Ruzurgi (amifampridine) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS)</h3>
<p>As the first treatment for adults with Lambert-Eaton Myasthenic Syndrome (LEMS), the 2018 FDA approval of MDA-supported treatment Firdapse marked a significant step forward in addressing the needs of individuals with LEMS by providing a dedicated therapy to improve muscle strength and quality of life.</p>
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<h2>2019</h2>
<h3>FDA approves Zolgensma (onasemnogenebeparvovec-xioi) for the treatment of SMA</h3>
<p>In 2019, the FDA approved Zolgensma, a first-of-its-kind, one-time gene therapy that transformed the treatment landscape for SMA. This groundbreaking gene therapy was made possible through the contributions of countless MDA researchers.</p>
<a href="https://mdaquest.org/fda-approves-avexis-zolgensma-for-treatment-of-spinal-muscular-atrophy-in-pediatric-patients/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2019</h2>
<h3>FDA approves Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy</h3>
<p>In 2019, the FDA approved Vyondys 53 for the treatment of Duchenne muscular dystrophy. The second ever exon-skipping, disease-modifying drug for DMD, this MDA-supported treatment builds on the momentum created by eteplirsen in developing effective solutions for the challenges of Duchenne.</p>
<a href="https://mdaquest.org/fda-approves-sarepta-therapeutics-vyondys-53-for-treatment-of-dmd-amenable-to-exon-53-skipping/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2019</h2>
<h3>MDA Hosts First Clinical and Scientific Conference</h3>
<p>In 2019, MDA united its previously separate Clinical and Scientific Conferences into one premier event, bringing together the world’s leading experts in neuromuscular medicine and research. This decision fostered interdisciplinary collaboration, facilitating the exchange of ideas between clinicians, researchers, and industry leaders. The combined conference has since become a prestigious convening of experts, advancing innovations in care, sharing groundbreaking research, and accelerating progress in the neuromuscular field. </p>
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<h2>2020</h2>
<h3>FDA approves Viltepso (viltolarsen) to treat Duchenne muscular dystrophy</h3>
<p>In 2020, the FDA approved the third-ever, exon-skipping treatment, Viltepso, for the fight against DMD. Supported by MDA, this breakthrough builds upon the successes of eteplirsen and golodirsen to create more meaningful treatment options for those living with Duchenne. </p>
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<h2>2020</h2>
<h3>FDA approves Evrysdi (risdiplam) to treat spinal muscular atrophy</h3>
<p>In 2020, the FDA approved Evrysdi, the third-ever disease modifying therapy approved to treat spinal muscular atrophy. As the leading genetic cause of infant death, this SMA treatment has had a resounding effect on people throughout the MDA community. </p>
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<h2>2020</h2>
<h3>Congress passes the ALS Disability Insurance Access Act</h3>
<p>In 2020, Congress passed the ALS Disability Insurance Access Act, helping address the urgent needs of the ALS community by eliminating the benefit waiting period and ensuring that individuals with ALS can access SSDI benefits immediately after approval, to receive timely assistance for medical expenses, adaptive equipment, and other essential needs. In partnership with several ALS organizations, MDA's advocacy work helped bring about this landmark legislation, furthering support for individuals and families during a challenging time.</p>
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<h2>2020</h2>
<h3>MDA launches Let's Play</h3>
<p>In 2020, MDA introduced the Let’s Play program, a unique initiative designed to build an inclusive community for gamers living with neuromuscular diseases. Through adaptive gaming technology, tournaments, and social connections, Let’s Play empowers individuals to participate in gaming experiences regardless of physical limitations.</p>
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<h2>2020</h2>
<h3>MDA introduces MDA Virtual Summer Camp</h3>
<p>In response to the COVID-19 pandemic, MDA reimagined its beloved Summer Camp by creating a virtual experience for kids and teens living with neuromuscular diseases. The Virtual Summer Camp brought the spirit of connection, fun, and independence directly into campers' homes through interactive activities, games, and shared experiences, ensuring the MDA Summer Camp tradition continued to provide meaningful opportunities for friendship and engagement.</p>
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<h2>2021</h2>
<h3>FDA approves Vyvgart (efgartigimod alfa-fcab) to treat generalized myasthenia gravis.</h3>
<p>In 2021, the FDA approved Vyvgart as a treatment for generalized myasthenia gravis. Replacing time-consuming infusions with a simple, at-home shot, this MDA-supported treatment creates new hope, new freedom, and new possibilities for those living with gMG. </p>
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<h2>2021</h2>
<h3>FDA approves Amondys 45 (casimersen) to treat Duchenne muscular dystrophy</h3>
<p>The FDA’s decision to approve Amondys 45 highlights the importance of years of commitment to supporting and funding breakthrough research by MDA and others into gene identification and unlocking the cause of DMD.</p>
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<h2>2021</h2>
<h3>FDA approves Octagam 10% [Immune Globulin Intraveneous (Human)] to treat adult dermatomyositis</h3>
<p>The FDA’s approval of Octapharma’s Octagam 10% [Immune Globulin Intravenous (Human)] for adults with dermatomyositis marked a meaningful step forward in treating inflammatory myopathies. MDA’s longstanding commitment to funding key research on muscle diseases helped build the knowledge base that makes treatments like Octagam 10 possible.</p>
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<h2>2021</h2>
<h3>FDA approves Nexviazyme (avalglucosidase alfa-ngpt) to treat late-onset Pompe disease</h3>
<p>The 2021 approval of Nexviazyme by the FDA provided new treatment options for people and families living with Pompe disease. An alternative therapy to the MDA-supported Myozyme, this approval marked an important expansion of drug options to improve lives within the Pompe and MDA communities. </p>
<a href="https://mdaquest.org/fda-approves-sanofi-genzymes-nexviazyme-for-treatment-of-pompe-disease/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2021</h2>
<h3>Congress passes the ACT for ALS</h3>
<p>In 2021, Congress passed the ACT for ALS, a landmark piece of legislation that accelerates access to urgently needed therapies for the ALS and neuromuscular disease communities. Supported by advocacy from MDA and allies, this Act establishes new pathways to fast-track experimental treatments and expands federal funding for ALS research, bringing hope to thousands of individuals and families facing these progressive diseases. </p>
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<h2>2021</h2>
<h3>MDA establishes the MDA Mentorship Program</h3>
<p>In 2021, MDA launched the MDA Mentorship Program to support youth and young adults with neuromuscular disease as they navigate higher education and career pathways. This unique program pairs participants with mentors who provide guidance, share insights, and foster connections that help young adults pursue their academic and professional goals with confidence. </p>
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<h2>2022</h2>
<h3>FDA approves Ultomiris (ravulizumab-cwvz) to treat generalized myasthenia gravis</h3>
<p>The 2022 FDA approval of Ultomiris for treating generalized myasthenia gravis welcomed new therapeutic options for those living with the disease. Studied across 36 MDA Care Centers, this drug and its approval signaled another major step forward in the fight against gMG. </p>
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<h2>2022</h2>
<h3>FDA approves Relyvrio (sodium phenylbutyrate/taurursodiol) to treat ALS</h3>
<p>In 2022, the FDA approval of Relyvrio marked the fourth approved therapy to treat ALS and the first to target its genetic cause. Developed with substantial support by MDA and other organizations, this treatment is a realization of the hope that genetic medicines could be effective for ALS patients and their families. </p>
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<h2>2022</h2>
<h3>MDA introduces the Quest Media Lifestyle platform</h3>
<p>In 2022, MDA introduced the Quest Media Lifestyle platform, a comprehensive resource designed to enhance the lives of individuals and families in the disability community. Through engaging articles, expert insights, personal stories, and practical advice, Quest Media offers readers a supportive space to explore topics ranging from health and wellness to accessible technology, education, and career growth. </p>
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<h2>2022</h2>
<h3>MDA Advocacy Collaboration Grant program launched</h3>
<p>Established in 2022, the Muscular Dystrophy Association Advocacy Collaboration Grant Program funds public policy and advocacy projects that benefit the neuromuscular disease community, supporting initiatives such as non-partisan advocacy campaigns, research projects, stakeholder meetings, and grassroots advocate training.</p>
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<h2>2022</h2>
<h3>The Centers for Medicare and Medicaid Services creates new diagnostic codes for LGMD</h3>
<p>In 2022, the Centers for Medicare and Medicaid services created new diagnostic codes for limb-girdle muscular dystrophy (LGMD), potentially shortening the diagnostic odyssey that patients face. Lobbied for by MDA and a number of LGMD organizations, these codes will help provide more precise medical care, improve clinical trials, and increase future access to targeted treatments. </p>
<a href="https://mdaquest.org/how-the-new-lgmd-diagnostic-codes-could-benefit-the-lgmd-community/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2022</h2>
<h3>Congress passes the FDA User Fee Reauthorization Act</h3>
<p>In 2022, the FDA User Fee Reauthorization Act passed Congress, clearing the way for an acceleration of treatment and cure development for rare diseases. This MDA-supported legislation included a suite of incredibly important programs and initiatives including expedited regulatory review, investment in gene and cell therapies, and more. </p>
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<h2>2023</h2>
<h3>FDA approves Agamree (vamorolone) to treat Duchenne muscular dystrophy</h3>
<p>In 2023, Agamree, a novel therapy supported by MDA Venture Philanthropy, was approved by the FDA for the treatment of DMD. </p>
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<h2>2023</h2>
<h3>FDA approves Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease</h3>
<p>This approval marked a new chapter in expanding treatment options for Pompe disease. MDA’s funding of foundational research at Duke University contributed to the development of Myozyme, the first approved Pompe therapy.</p>
<a href="https://www.mda.org/disease/metabolic-myopathies/types/acid-maltase-deficiency-pompe-disease" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2023</h2>
<h3>FDA approves Rystiggo (rozanolixizumab-noli) to treat generalized myasthenia gravis</h3>
<p>Rystiggo (rozanolixizumab-noli) is used to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive.</p>
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<h2>2023</h2>
<h3>FDA approves Qalsody (tofersen) to treat ALS</h3>
<p>Qalsody is approved for use for adults with an ALS SOD1 mutation.</p>
<a href="https://mdaquest.org/fda-approves-biogens-qalsody-for-treatment-of-sod1-als/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2023</h2>
<h3>FDA approves Skyclaris (omaveloxolone) to treat Friederich's ataxia</h3>
<p>Clinical trials of Skyclarys took place at MDA Care Center Network locations including UCLA, University of Florida Neurology, Emory University Hospital, The Ohio State University, and Children's Hospital of Philadelphia.</p>
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<h2>2023</h2>
<h3>MDA Kickstart Program launched</h3>
<p>In 2023, MDA introduced the Kickstart Program to accelerate the development of therapies for ultra-rare neuromuscular diseases. By providing critical funding and resources to early-stage projects, Kickstart bridges the gap between basic research and clinical application, paving the way for innovative treatments by fostering collaboration among researchers.</p>
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<h2>2023</h2>
<h3>MDA creates the Gene Therapy Support Network</h3>
<p>In 2023, MDA launched the Gene Therapy Support Network to equip individuals and families with essential information and support as groundbreaking gene therapies emerge for neuromuscular diseases. This first-of-its-kind initiative offers up-to-date resources, expert guidance, and a dedicated support system to help families navigate the complex world of gene therapy. </p>
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<h2>2023</h2>
<h3>MDA establishes MDA Connect</h3>
<p>In 2023, MDA introduced the MDA Connect program, offering individuals and families affected by neuromuscular diseases personalized, one-on-one video consultations with MDA Specialists. This program provides convenient access to expert guidance on navigating care within MDA’s Care Center Network, locating resources, and learning about MDA programs and engagement opportunities. </p>
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<h2>2023</h2>
<h3>MDA Introduces Family Getaways Program </h3>
<p>In 2023, MDA launched the MDA Family Getaways program, offering MDA community members of all ages, along with their families, unique and accessible recreation experiences and connection to others. </p>
<a href="https://www.mda.org/family-getaways" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2024</h2>
<h3>A new era of treatment for neuromuscular diseases emerges</h3>
<p>In 2024, a new era of treatments emerged with more than 20 FDA-approved therapies now available for neuromuscular diseases—including the first ever treatments for Duchenne muscular dystrophy and spinal muscular atrophy—thanks in part to research supported by MDA</p>
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<h2>2024</h2>
<h3>FDA approves Duvyzat (givinostat) to treat Duchenne muscular dystrophy</h3>
<p>In 2024, MDA's funding of foundational research led to the FDA approval of Duvyzat, a drug for treating DMD in people 6 years and older. </p>
<a href="https://mdaquest.org/fdas-approval-of-duvyzat-givinostat-offers-new-hope-to-people-living-with-duchenne-muscular-dystrophy/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></span></button></a>
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<h2>2024</h2>
<h3>MDA Gene Therapy Support Network honored at 2024 Advanced Therapies Awards</h3>
<p>MDA’s Gene Therapy Support Network received honors at the 2024 Patient Advocacy Award for Non-Profits at the Advanced Therapies Awards.</p>
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<h2>2024</h2>
<h3>Congress Passes Air Travel Accessibility Reforms</h3>
<p>Due to the dedicated efforts of advocates and the MDA community, Congress passed landmark air travel accessibility reforms within the FAA Reauthorization Act of 2024. This historic legislation mandated critical improvements in air travel standards to ensure safe, dignified, and accessible travel for persons with disabilities. Additionally, it addressed long-standing barriers faced by people with mobility impairments, setting new standards for seating, boarding procedures, and wheelchair handling, among other areas. </p>
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<h2>2024</h2>
<h3>All 50 states and DC implement newborn screening for SMA</h3>
<p>Thanks in part to MDA’s strong advocacy efforts, all 50 states and Washington, DC, implemented newborn screening for spinal muscular atrophy (SMA) in 2024, making early diagnosis and intervention possible for every newborn with this condition.</p>
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<h2>2024</h2>
<h3>MDA College Scholarship Program Established</h3>
<p>In 2024, MDA introduced a college scholarship program designed to empower young people with neuromuscular disease as they pursue higher education and career training. This program provides financial support to students, helping to alleviate barriers to educational opportunities and open pathways to personal and professional growth.</p>
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<h3>The legacy of<br /> a community committed</h3>
<p>Research. Policy. Opportunity. Freedom. <br />As we celebrate 75 years of progress, we have no intention of slowing down. We’re building on this momentum by:</p>
<ul>
<li>Expanding research into gene therapies and muscle regeneration</li>
<li>Advocating for policies that empower people with neuromuscular disease</li>
<li>Supporting access to medical care through MDA’s Care Center Network</li>
<li>Continuing to build and empower a strong, connected community</li>
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<p>Scroll down to explore the moments in which we’ve made history—and the impacts that have been felt by millions.</p>
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<h2>1949</h2>
<h3>Dr. Ade Milhorat is one of few doctors researching muscular dystrophies worldwide</h3>
<p>In 1949, Dr. Ade Milhorat of New York Hospital-Cornell Medical Center was one of only a handful of doctors in the world researching muscular dystrophies. His research work lost funding, and it might well have been the end of his efforts were it not for a friend who also had muscular dystrophy. That friend was Paul Cohen.</p>
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<h2>1950</h2>
<h3>The Muscular Dystrophy Association of America is founded</h3>
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<p>On June 6, 1950, Paul Cohen, a prominent New York businessman with a form of muscular dystrophy (FSH), invited a group of individuals to meet in his Rye, NY office. Each had a personal connection to muscular dystrophy, and the gathering focused on the urgent need to raise funds to advance research seeking treatments and cures for muscular dystrophy. The group formed the Muscular Dystrophy Association of America (MDAA)*, and two months later, MDA made its first research grant to support Dr. Milhorat’s research program.</p>
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<h2>1952</h2>
<h3>Dean Martin, Jerry Lewis promote MDA on national radio</h3>
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<p>During their January 4, 1952 radio program, Martin and Lewis make an appeal for nationwide support of MDA. Later that year, Martin and Lewis were named MDA National Co-Chairmen.</p>
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<h2>1952</h2>
<h3>MDA Ambassador Program launched</h3>
</span>
<p>MDA introduces its first Ambassador Program to raise awareness about muscular dystrophy and other neuromuscular disorders. Over time, the program evolves to highlight the strength and diverse stories of people living with neuromuscular disease in the MDA community.</p>
<a href="https://www.mda.org/ambassadors" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1952</h2>
<h3>Five MDA Local Telethons Held in Two Years</h3>
</span>
<p>Early to recognize the power of television to bolster awareness and raise income for the Association, MDA’s top fundraising priority was the pioneering work of establishing local Telethons featuring a variety of celebrities. Thanks to the help of top stars like Robert Alda, Dick Van Dyke, Captain Video and Virginia Graham, MDA successfully hosted five local Telethons in two years in Cleveland; Washington DC; Atlanta; Grand Rapids; and Madison, Wisconsin.</p>
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<span class="green">
<h2>1952</h2>
<h3>The International Association Resolves to Fight Muscular Dystrophy and "Fills the Boot"</h3>
</span>
<p>Early in 1952, a group of families affected by muscular dystrophy approached Local 718 fire station in the Boston area to ask professional fire fighters to help fight muscular dystrophy. Responding enthusiastically, the fire fighters took to the streets with their boots in hand to ask greater Boston to make donations that would be used to fight muscular dystrophy. The Fill the Boot campaign was an instant success and on August 19, 1954, the International Association of Fire Fighters (IAFF) membership passed a resolution to support the Association’s fight against muscular dystrophy until treatments and cures are found.</p>
<a href="https://www.mda.org/get-involved/meet-our-partners/international-association-fire-fighters" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1953</h2>
<h3>The first MDA Care Centers are established</h3>
</span>
<p>The first MDA Care Centers are established at NYU Langone Health and University of Rochester Medical Center, providing specialized neuromuscular disease support and care—a foundation of MDA’s Care Center Network.</p>
<a href="https://www.mda.org/care/mda-care-centers" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1953</h2>
<h3>National Association of Letter Carriers becomes MDA's first national sponsor</h3>
</span>
<p>MDA Founder Paul Cohen recruits the National Association of Letter Carriers (NALC) as the organization’s first national sponsor. The NALC quickly established a nationwide door-to-door campaign for MDA, the first of which took place on November 25, 1953. The inaugural campaign was heralded by a special two-hour coast to coast television show hosted by Martin and Lewis.</p>
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<span class="green">
<h2>1955</h2>
<h3>Eleanor Gehrig named MDA's National Campaign Chairperson
</h3>
</span>
<p>After losing her husband, New York Yankees icon Lou Gehrig, to ALS, Eleanor Gehrig made it her mission to help others with the disease. To do so, she partnered with MDA, spearheading advocacy and care efforts that changed the future of life with ALS for good.</p>
<a href="https://www.youtube.com/watch?v=ksH7zyxBnaA" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1955</h2>
<h3>MDA holds first MDA Summer Camp</h3>
</span>
<p>MDA holds its first Summer Camp with 16 campers in Sussex, NJ, creating a unique place for children with neuromuscular diseases to experience independence, build friendships, and have fun in an accessible environment.</p>
<a href="https://www.mda.org/summer-camp" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1956</h2>
<h3>Martin and Lewis Local Telethon for MDA at Carnegie Hall</h3>
</span>
<p>Martin and Lewis co-hosted the 1956 Telethon at New York City’s famed Carnegie Hall and broadcast June 29-30 on Dumont Station WABD (now WABC) in New York, raising some $600,000. </p>
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<span class="blue">
<h2>1966</h2>
<h3>MDA's Legendary Labor Day Telethon Series Begins</h3>
</span>
<p> The first MDA Jerry Lewis Labor Day Telethon was broadcast in 1966 by a single station, WNEW-TV in New York. The telecast, which resulted in $1,002,114 being pledged, was so successful that MDA selected Labor Day weekend for its future Telethons.</p>
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<h2>1971</h2>
<h3>MDA Labor Day Telethon seen across the country</h3>
<p>Robert M. Bennett (President of Metromedia Broadcasting), creates the “Love Network” of stations that facilitates the nation’s first networked Telethon in 1971.</p>
<a href="https://www.mda.org/about-mda/history" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1983</h2>
<h3>The Orphan Drug Act (ODA) of 1983 enacted.</h3>
</span>
<p>The Orphan Drug Act (ODA) of 1983 was enacted to provide incentives to support research and development into drugs for rare diseases with small patient populations.</p>
<a href="https://www.mda.org/get-involved/advocacy" target="_blank"><button class="noselect banner2"><span class="text">Get Involved</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1986</h2>
<h3>CITGO Joins Forces with MDA</h3>
<span>
<p>CITGO Petroleum Corporation begins its enduring partnership with the Muscular Dystrophy Association. Over the decades, CITGO employees, marketers, and retailers have supported MDA through volunteer efforts and community engagement, helping to advance critical programs such as MDA Care Centers, summer camps, and research initiatives.</p>
<a href="https://www.mda.org/get-involved/meet-our-partners/citgo-petroleum-corporation" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<!-- 1986-B -->
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<span class="green">
<h2>1986</h2>
<h3>MDA-funded discovery of the DMD gene launches a new era in genetic research and therapies.</h3>
</span>
<p>In 1986, MDA-funded researcher Dr. Lou Kunkel and his team identified the gene responsible for Duchenne and Becker muscular dystrophy, a landmark discovery that paved the way for targeted research and innovative treatments. This breakthrough marked the beginning of the gene discovery era, transforming scientific understanding of neuromuscular diseases and laying the foundation for advancements in genetic therapies that continue to benefit the neuromuscular community today.</p>
<a href="https://www.mda.org/science/what-weve-achieved" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>1988</h2>
<h3>MDA funds SMA Cell Bank to advance gene discovery</h3>
</span>
<p>MDA funds Dr. Conrad Gilliam and collaborators to establish the SMA Cell Bank, accelerating the discovery of the SMA-causing gene through groundbreaking natural history studies.</p>
<a href="https://www.mda.org/science/research-grants" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>1990</h2>
<h3>Americans with Disabilities Act (ADA) signed into law</h3>
</span>
<p>On July 26, 1990, President George H.W. Bush signed into law this landmark civil rights legislation intended to eliminate barriers for people with disabilities. Driven by the advocacy work of MDA and other organizations, this pivotal law represents a critical milestone in advancing the rights and independence of people living with neuromuscular diseases.</p>
<a href="https://www.votervoice.net/MDA/1/campaigns" target="_blank"><button class="noselect banner2"><span class="text">Get Involved</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>1992</h2>
<h3>Genetic mutations causing myotonic dystrophy type 1 identified</h3>
</span>
<p>In 1992, MDA supported research leading to the identification of genetic mutations causing myotonic dystrophy type 1 (DM1)</p>
<a href="https://www.mda.org/disease" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h2><br />1993</h2>
<h3>Mutations in PMP22 identified as a causative for CMT1A</h3>
<span>
<p>In 1993, mutations in PMP22 were identified as a causative for CMT1A (first localized to 17p11.2) by a collaborative group sponsored by MDA.</p>
<a href="https://www.mda.org/science/grants-at-a-glance" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>1995</h2>
<h3>FDA approves Riluzole, first treatment for ALS</h3>
</span>
<p>In 1995, the FDA approved riluzole, the first-ever treatment for ALS, offering new hope for people living with the disease. Backed by years of research, including contributions from MDA-supported scientists, riluzole’s approval represented a critical step in ALS treatment, paving the way for further advancements in therapies aimed at improving quality of life and extending survival for people living with ALS.</p>
<a href="https://www.mda.org/disease/amyotrophic-lateral-sclerosis" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>1996</h2>
<h3>American Medical Association honors MDA with the Lifetime Achievement Award</h3>
</span>
<p>In 1996, the American Medical Association honored MDA with the prestigious Lifetime Achievement Award, recognizing decades of pioneering work in neuromuscular disease research, care, and advocacy. This accolade underscores MDA's enduring commitment to leading efforts that have shaped the landscape of neuromuscular medicine.</p>
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<span class="blue">
<h2>1998</h2>
<h3>MDA-funded work leads to three FDA-approved exon skipping drugs for Duchenne muscular dystrophy</h3>
</span>
<p>In 1998. MDA-funded research contributed to the development of three FDA-approved exon-skipping drugs for Duchenne muscular dystrophy, offering targeted treatment options for specific genetic mutations within the disorder. These therapies marked a significant advancement in Duchenne care, slowing disease progression for eligible individuals and opening doors to further genetic-based approaches.</p>
<a href="https://www.mda.org/science/creating-a-new-therapy" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>1999</h2>
<h3>MDA funds first clinical trial for gene therapy for any type of muscular dystrophy</h3>
</span>
<p>In 1999, the Muscular Dystrophy Association funded the first clinical trial for gene therapy for any type of muscular dystrophy. This trial was led by Dr. Jerry Mendell and was the first gene therapy trial for muscular dystrophy.</p>
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<h2>2000</h2>
<h3>MDA funds research to boost SMN2 gene expression for SMA treatment.</h3>
<p>In 2000, MDA funded Dr. Christian Lorson (Arizona State University) to investigate factors that can help increase expression of full length SMN protein from the SMN2 gene. </p>
<a href="https://www.mda.org/care/gene-therapy-community-support" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2001</h2>
<h3>Genetic mutation causing myotonic dystrophy type 2 identified.</h3>
<p>In 2001, a team led by Dr. Thomas R. Ranum and Dr. Maurice Day discovered the CCTG tetranucleotide repeat expansion in the CNBP (ZNF9) gene responsible for DM2. </p>
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<h2>2001</h2>
<h3>U.S. Congress Passes the MD-CARE Act</h3>
<p>The 2001 passage of the MD CARE Act, championed by MDA advocates and allies, marked a pivotal achievement in the fight against muscular dystrophy by expanding federal support for research, care, and resources across the country. </p>
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<h2>2003</h2>
<h3>The Centers of Excellence program in muscular dystrophy research is established by NIH</h3>
<p>The Centers of Excellence program in muscular dystrophy research was established in 2003 by NIH in honor of the late Senator Paul D. Wellstone of Minnesota. </p>
<p>The Wellstone Centers focus on translating basic research into clinical applications, enhancing diagnostics, and developing new therapies, with an emphasis on collaboration across institutions. </p>
<a href="https://www.mda.org/care/visiting-an-mda-care-center" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2006</h2>
<h3>MDA funds the first-ever gene therapy trial for Duchenne muscular dystrophy</h3>
<p>In 2006, MDA funded the first-ever gene therapy trial for Duchenne muscular dystrophy, marking a transformative moment in the pursuit of neuromuscular disease treatments. This pioneering trial paveed the way for gene-based approaches aimed at addressing the root causes of Duchenne, opening the door to innovative therapies for a range of neuromuscular diseases. </p>
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<h2>2006</h2>
<h3>FDA approves Lumizyme (alglucosidase alfa) for the treatment of Pompe Disease</h3>
<p>In 2006, the FDA approved Lumizyme, a groundbreaking enzyme replacement therapy for Pompe disease. The development of the therapy, funded in part by MDA, marked a significant advancement in care, offering new hope for improved quality of life.</p>
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<!-- 2007 -->
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<h2>2007</h2>
<h3>MDA funds Dr. Adrian Krainer's antisense therapy research, leading to Spinraza for SMA</h3>
<p>In 2007, MDA funded pioneering research by Dr. Adrian Krainer in antisense therapy, leading to a breakthrough in treatment for spinal muscular atrophy (SMA). Dr. Krainer’s work culminates in the development and FDA approval of Spinraza, the first therapy to effectively target the genetic root of SMA, transforming SMA from a terminal diagnosis in young children to a manageable condition.</p>
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<span class="green">
<h2>2010</h2>
<h3>Surveying New Therapeutive Avenues</h3>
</span>
<p>In 2010, MDA awarded multiple grants to explore SMA gene therapy, gene delivery, novel ASO therapies, and new small molecule targets in mouse and zebrafish models of SMA.</p>
<a href="https://www.mda.org/science/grants-at-a-glance" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>2011</h2>
<h3>Identification of C9ORF mutations in ALS</h3>
</span>
<p>In 2011, MDA-funded research led to the identification of C9ORF72 gene mutation as the most common genetic cause of ALS and FTD. </p>
<a href="https://www.mda.org/disease/amyotrophic-lateral-sclerosis" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2015</h2>
<h3>MDA makes the difficult decision to end its historic Telethon tradition</h3>
</span>
<p>As families and supporters began looking for new ways to support and get involved with the organization, MDA embraced new opportunities through social media and other digital channels to inspire the nation in support of the fight against neuromuscular diseases.</p>
<a href="https://www.mda.org/get-involved/participate-in-an-event" target="_blank"><button class="noselect banner2"><span class="text">Get Involved</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>2015</h2>
<h3>FDA approves Keveyis for treating periodic paralysis</h3>
</span>
<p>In 2015, the FDA approved MDA-funded Keveyis (Dichlorphena-mide) for the treatment of hyperkalemic and hypokalemic periodic paralysis.</p>
<a href="https://www.mda.org/science/funding-opportunities" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2015</h2>
<h3>MDA establishes the MDA Resource Center to provide trusted guidance and support nationwide</h3>
</span>
<p>In 2015, MDA established the MDA Resource Center as the organization’s national information hub, offering individuals and families a reliable source of information, guidance, and support to navigate life with neuromuscular disease. </p>
<a href="https://www.mda.org/care/mda-resource-center" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="green">
<h2>2016</h2>
<h3>FDA approves Exondys 51 (eteplirsen) for the treatment of DMD</h3>
</span>
<p>After decades of funding Duchenne muscular dystrophy research, the MDA-supported landmark treatment, Exondys 51 (eteplirsen), was approved by the FDA in 2016. This therapy was the first approved drug to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. </p>
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<span class="green">
<h3><br />FDA approves Spinraza (nusinersen) for the treatment of SMA</h3>
</span>
<p>In 2016, the FDA issued a landmark approval for Spinraza, providing life-changing therapeutic options for indivisuals with SMA. With foundational support from MDA, this treatment offers the potential to slow disease progression and improve motor function. </p>
<a href="https://mdaquest.org/a-revolutionary-decade-for-spinal-muscular-atrophy-therapies/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2017</h2>
<h3>FDA approves Emflaza (deflazacort) for the treatment of Duchenne Muscular Dystrophy</h3>
</span>
<p>In 2017, the FDA approved Emflaza, a signature treatment for the most common form of childhood muscular dystrophy, Duchenne, after decades of MDA research, funding, and trials. </p>
<a href="https://www.mda.org/press-releases/mda-celebrates-news-fda-approval-emflaza-treatment-duchenne-muscular-dystrophy" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="green">
<h3><br />FDA approves Radicava (edaravone) for the treatment of ALS</h3>
</span>
<p>After completing human trials at more than a dozen MDA care centers, the FDA approved Radicava for the treatment of ALS in 2017. </p>
<span class="green">
<h3><br />FDA approves Soliris (eculizumab) for the treatment of myasthenia gravis</h3>
</span>
<p>In 2017, the FDA approved Soliris for the treatment of myasthenia gravis. A first-of-its-kind complement inhibitor drug, this therapeutic strategy has paved the way for new care options for the MDA community, and new paths for MDA-funded research. </p>
<a href="https://www.mda.org/disease/myasthenia-gravis/research" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2018</h2>
<h3>Decoding Disease Mechanisms</h3>
</span>
<p>In 2018, MDA funded grants to Dr. Arthur Burghes (Ohio State) for identifying modifiers of disease and Dr. Rashmi Kothary(Ottawa Hospital Research Institute) to study abnormal metabolism in SMA. </p>
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<h2>2018</h2>
<h3>MDA launches the MOVR Data Hub™</h3>
</span>
<p>In 2018, MDA launched the MOVR (NeuroMuscular ObserVational Research) Data Hub™, a neuromuscular disease data platform that centralizes clinical information from MDA Care Centers across the country. This comprehensive resource empowers researchers, clinicians, and healthcare providers with real-time insights, accelerating progress in understanding disease patterns, treatment outcomes, and patient needs. </p>
<a href="https://www.mda.org/science/movr" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2018</h2>
<h3>Adrian Krainer, PhD, wins Breakthrough Prize for developing Spinraza, an SMA therapy</h3>
</span>
<p>In 2018, MDA researcher Adrian Krainer, PhD, received the Breakthrough Prize in Life Sciences for the development of an effective antisense oligonucleotide therapy (Spinraza) for children with SMA.Dr. Krainer’s work with Spinraza underscores the power of targeted genetic therapies and sets a precedent for future innovations in treating neuromuscular diseases.</p>
<a href="https://mdaquest.org/how-mda-invests-in-research-success/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2018/2019</h2>
<h3>FDA approves Firdapse/Ruzurgi (amifampridine) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS)</h3>
</span>
<p>As the first treatment for adults with Lambert-Eaton Myasthenic Syndrome (LEMS), the 2018 FDA approval of MDA-supported treatment Firdapse marked a significant step forward in addressing the needs of individuals with LEMS by providing a dedicated therapy to improve muscle strength and quality of life.</p>
<a href="https://www.mda.org/disease/lambert-eaton-myasthenic-syndrome/research" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2019</h2>
<h3>FDA approves Zolgensma (onasemnogenebeparvovec-xioi) for the treatment of SMA</h3>
</span>
<p>In 2019, the FDA approved Zolgensma, a first-of-its-kind, one-time gene therapy that transformed the treatment landscape for SMA. This groundbreaking gene therapy was made possible through the contributions of countless MDA researchers.</p>
<a href="https://mdaquest.org/fda-approves-avexis-zolgensma-for-treatment-of-spinal-muscular-atrophy-in-pediatric-patients/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2019</h2>
<h3>FDA approves Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy</h3>
</span>
<p>In 2019, the FDA approved Vyondys 53 for the treatment of Duchenne muscular dystrophy. The second ever exon-skipping, disease-modifying drug for DMD, this MDA-supported treatment builds on the momentum created by eteplirsen in developing effective solutions for the challenges of Duchenne.</p>
<a href="https://mdaquest.org/fda-approves-sarepta-therapeutics-vyondys-53-for-treatment-of-dmd-amenable-to-exon-53-skipping/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2019</h2>
<h3>MDA Hosts First Clinical and Scientific Conference</h3>
</span>
<p>In 2019, MDA united its previously separate Clinical and Scientific Conferences into one premier event, bringing together the world’s leading experts in neuromuscular medicine and research. This decision fostered interdisciplinary collaboration, facilitating the exchange of ideas between clinicians, researchers, and industry leaders. The combined conference has since become a prestigious convening of experts, advancing innovations in care, sharing groundbreaking research, and accelerating progress in the neuromuscular field. </p>
<a href="https://www.mda.org/conferences/2019-clinical-and-scientific-conference" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2020</h2>
<h3>FDA approves Viltepso (viltolarsen) to treat Duchenne muscular dystrophy</h3></span>
<p>In 2020, the FDA approved the third-ever, exon-skipping treatment, Viltepso, for the fight against DMD. Supported by MDA, this breakthrough builds upon the successes of eteplirsen and golodirsen to create more meaningful treatment options for those living with Duchenne. </p>
<span class="blue">
<h3><br />FDA approves Evrysdi (risdiplam) to treat spinal muscular atrophy</h3></span>
<p>In 2020, the FDA approved Evrysdi, the third-ever disease modifying therapy approved to treat spinal muscular atrophy. As the leading genetic cause of infant death, this SMA treatment has had a resounding effect on people throughout the MDA community. </p>
<a href="https://mdaquest.org/fda-approves-genentechs-evrysdi-for-treatment-of-sma-in-pediatric-and-adult-patients/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2020</h2>
<h3>Congress passes the ALS Disability Insurance Access Act</h3></span>
<p>In 2020, Congress passed the ALS Disability Insurance Access Act, helping address the urgent needs of the ALS community by eliminating the benefit waiting period and ensuring that individuals with ALS can access SSDI benefits immediately after approval, to receive timely assistance for medical expenses, adaptive equipment, and other essential needs. In partnership with several ALS organizations, MDA's advocacy work helped bring about this landmark legislation, furthering support for individuals and families during a challenging time.</p>
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<h2>2020</h2>
<h3>MDA launches Let's Play</h3></span>
<p>In 2020, MDA introduced the Let’s Play program, a unique initiative designed to build an inclusive community for gamers living with neuromuscular diseases. Through adaptive gaming technology, tournaments, and social connections, Let’s Play empowers individuals to participate in gaming experiences regardless of physical limitations.</p>
<a href="https://www.mda.org/lets-play" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2020</h2>
<h3>MDA introduces MDA Virtual Summer Camp</h3></span>
<p>In response to the COVID-19 pandemic, MDA reimagined its beloved Summer Camp by creating a virtual experience for kids and teens living with neuromuscular diseases. The Virtual Summer Camp brought the spirit of connection, fun, and independence directly into campers' homes through interactive activities, games, and shared experiences, ensuring the MDA Summer Camp tradition continued to provide meaningful opportunities for friendship and engagement.</p>
<a href="https://www.mda.org/virtual-camp" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2021</h2>
<h3>FDA approves Vyvgart (efgartigimod alfa-fcab) to treat generalized myasthenia gravis.</h3></span>
<p>In 2021, the FDA approved Vyvgart as a treatment for generalized myasthenia gravis. Replacing time-consuming infusions with a simple, at-home shot, this MDA-supported treatment creates new hope, new freedom, and new possibilities for those living with gMG. </p>
<span class="blue">
<h3><br />FDA approves Amondys 45 (casimersen) to treat Duchenne muscular dystrophy</h3></span>
<p>The FDA’s decision to approve Amondys 45 highlights the importance of years of commitment to supporting and funding breakthrough research by MDA and others into gene identification and unlocking the cause of DMD.</p>
<span class="blue">
<h3><br />FDA approves Octagam 10% [Immune Globulin Intraveneous (Human)] to treat adult dermatomyositis</h3></span>
<p>The FDA’s approval of Octapharma’s Octagam 10% [Immune Globulin Intravenous (Human)] for adults with dermatomyositis marked a meaningful step forward in treating inflammatory myopathies. MDA’s longstanding commitment to funding key research on muscle diseases helped build the knowledge base that makes treatments like Octagam 10 possible.</p>
<a href="https://www.mda.org/disease/dermatomyositis" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h3><br />FDA approves Nexviazyme (avalglucosidase alfa-ngpt) to treat late-onset Pompe disease</h3></span>
<p>The 2021 approval of Nexviazyme by the FDA provided new treatment options for people and families living with Pompe disease. An alternative therapy to the MDA-supported Myozyme, this approval marked an important expansion of drug options to improve lives within the Pompe and MDA communities. </p>
<a href="https://mdaquest.org/fda-approves-sanofi-genzymes-nexviazyme-for-treatment-of-pompe-disease/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2021</h2>
<h3>Congress passes the ACT for ALS</h3></span>
<p>In 2021, Congress passed the ACT for ALS, a landmark piece of legislation that accelerates access to urgently needed therapies for the ALS and neuromuscular disease communities. Supported by advocacy from MDA and allies, this Act establishes new pathways to fast-track experimental treatments and expands federal funding for ALS research, bringing hope to thousands of individuals and families facing these progressive diseases. </p>
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<h2>2021</h2>
<h3>MDA establishes the MDA Mentorship Program</h3></span>
<p>In 2021, MDA launched the MDA Mentorship Program to support youth and young adults with neuromuscular disease as they navigate higher education and career pathways. This unique program pairs participants with mentors who provide guidance, share insights, and foster connections that help young adults pursue their academic and professional goals with confidence. </p>
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<h2>2022</h2>
<h3>FDA approves Ultomiris (ravulizumab-cwvz) to treat generalized myasthenia gravis</h3></span>
<p>The 2022 FDA approval of Ultomiris for treating generalized myasthenia gravis welcomed new therapeutic options for those living with the disease. Studied across 36 MDA Care Centers, this drug and its approval signaled another major step forward in the fight against gMG. </p>
<a href="https://www.mda.org/disease/myasthenia-gravis" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h3><br />FDA approves Relyvrio (sodium phenylbutyrate/taurursodiol) to treat ALS</h3></span>
<p>In 2022, the FDA approval of Relyvrio marked the fourth approved therapy to treat ALS and the first to target its genetic cause. Developed with substantial support by MDA and other organizations, this treatment is a realization of the hope that genetic medicines could be effective for ALS patients and their families. </p>
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<h2>2022</h2>
<h3>MDA introduces the Quest Media Lifestyle platform</h3></span>
<p>In 2022, MDA introduced the Quest Media Lifestyle platform, a comprehensive resource designed to enhance the lives of individuals and families in the disability community. Through engaging articles, expert insights, personal stories, and practical advice, Quest Media offers readers a supportive space to explore topics ranging from health and wellness to accessible technology, education, and career growth. </p>
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<span class="blue">
<h2>2022</h2>
<h3>MDA Advocacy Collaboration Grant program launched</h3></span>
<p>Established in 2022, the Muscular Dystrophy Association Advocacy Collaboration Grant Program funds public policy and advocacy projects that benefit the neuromuscular disease community, supporting initiatives such as non-partisan advocacy campaigns, research projects, stakeholder meetings, and grassroots advocate training.</p>
<a href="https://www.votervoice.net/MDA/Blog" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>2022</h2>
<h3>The Centers for Medicare and Medicaid Services creates new diagnostic codes for LGMD</h3></span>
<p>In 2022, the Centers for Medicare and Medicaid services created new diagnostic codes for limb-girdle muscular dystrophy (LGMD), potentially shortening the diagnostic odyssey that patients face. Lobbied for by MDA and a number of LGMD organizations, these codes will help provide more precise medical care, improve clinical trials, and increase future access to targeted treatments. </p>
<a href="https://mdaquest.org/how-the-new-lgmd-diagnostic-codes-could-benefit-the-lgmd-community/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2022</h2>
<h3>Congress passes the FDA User Fee Reauthorization Act</h3></span>
<p>In 2022, the FDA User Fee Reauthorization Act passed Congress, clearing the way for an acceleration of treatment and cure development for rare diseases. This MDA-supported legislation included a suite of incredibly important programs and initiatives including expedited regulatory review, investment in gene and cell therapies, and more. </p>
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<span class="blue">
<h2>2023</h2>
<h3>FDA approves Agamree (vamorolone) to treat Duchenne muscular dystrophy</h3></span>
<p>In 2023, Agamree, a novel therapy supported by MDA Venture Philanthropy, was approved by the FDA for the treatment of DMD. </p>
<span class="blue">
<h3><br />FDA approves Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease</h3></span>
<p>This approval marked a new chapter in expanding treatment options for Pompe disease. MDA’s funding of foundational research at Duke University contributed to the development of Myozyme, the first approved Pompe therapy.</p>
<a href="https://www.mda.org/disease/metabolic-myopathies/types/acid-maltase-deficiency-pompe-disease" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h3><br />FDA approves Rystiggo (rozanolixizumab-noli) to treat generalized myasthenia gravis</h3></span>
<p>Rystiggo (rozanolixizumab-noli) is used to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive.</p>
<span class="blue">
<h3><br />FDA approves Qalsody (tofersen) to treat ALS</h3></span>
<p>Qalsody is approved for use for adults with an ALS SOD1 mutation.</p>
<a href="https://mdaquest.org/fda-approves-biogens-qalsody-for-treatment-of-sod1-als/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h3><br />FDA approves Skyclaris (omaveloxolone) to treat Friederich's ataxia</h3></span>
<p>Clinical trials of Skyclarys took place at MDA Care Center Network locations including UCLA, University of Florida Neurology, Emory University Hospital, The Ohio State University, and Children's Hospital of Philadelphia.</p>
<a href="https://www.mda.org/disease/friedreichs-ataxia" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>2023</h2>
<h3>MDA Kickstart Program launched</h3></span>
<p>In 2023, MDA introduced the Kickstart Program to accelerate the development of therapies for ultra-rare neuromuscular diseases. By providing critical funding and resources to early-stage projects, Kickstart bridges the gap between basic research and clinical application, paving the way for innovative treatments by fostering collaboration among researchers.</p>
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<span class="blue">
<h2>2023</h2>
<h3>MDA creates the Gene Therapy Support Network</h3></span>
<p>In 2023, MDA launched the Gene Therapy Support Network to equip individuals and families with essential information and support as groundbreaking gene therapies emerge for neuromuscular diseases. This first-of-its-kind initiative offers up-to-date resources, expert guidance, and a dedicated support system to help families navigate the complex world of gene therapy. </p>
<a href="https://www.mda.org/care/gene-therapy-community-support" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
<span class="blue">
<h3><br />MDA establishes MDA Connect</h3></span>
<p>In 2023, MDA introduced the MDA Connect program, offering individuals and families affected by neuromuscular diseases personalized, one-on-one video consultations with MDA Specialists. This program provides convenient access to expert guidance on navigating care within MDA’s Care Center Network, locating resources, and learning about MDA programs and engagement opportunities. </p>
<a href="https://www.mda.org/care/connect" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>2023</h2>
<h3>MDA Introduces Family Getaways Program </h3></span>
<p>In 2023, MDA launched the MDA Family Getaways program, offering MDA community members of all ages, along with their families, unique and accessible recreation experiences and connection to others. </p>
<a href="https://www.mda.org/family-getaways" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>2024</h2>
<h3>A new era of treatment for neuromuscular diseases emerges</h3></span>
<p>In 2024, a new era of treatments emerged with more than 20 FDA-approved therapies now available for neuromuscular diseases—including the first ever treatments for Duchenne muscular dystrophy and spinal muscular atrophy—thanks in part to research supported by MDA</p>
<span class="blue">
<h3><br />FDA approves Duvyzat (givinostat) to treat Duchenne muscular dystrophy</h3></span>
<p>In 2024, MDA's funding of foundational research led to the FDA approval of Duvyzat, a drug for treating DMD in people 6 years and older. </p>
<a href="https://mdaquest.org/fdas-approval-of-duvyzat-givinostat-offers-new-hope-to-people-living-with-duchenne-muscular-dystrophy/" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<span class="blue">
<h2>2024</h2>
<h3>MDA Gene Therapy Support Network honored at 2024 Advanced Therapies Awards</h3></span>
<p>MDA’s Gene Therapy Support Network received honors at the 2024 Patient Advocacy Award for Non-Profits at the Advanced Therapies Awards.</p>
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<span class="blue">
<h2>2024</h2>
<h3>Congress Passes Air Travel Accessibility Reforms</h3></span>
<p>Due to the dedicated efforts of advocates and the MDA community, Congress passed landmark air travel accessibility reforms within the FAA Reauthorization Act of 2024. This historic legislation mandated critical improvements in air travel standards to ensure safe, dignified, and accessible travel for persons with disabilities. Additionally, it addressed long-standing barriers faced by people with mobility impairments, setting new standards for seating, boarding procedures, and wheelchair handling, among other areas. </p>
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<span class="blue">
<h2>2024</h2>
<h3>All 50 states and DC implement newborn screening for SMA</h3></span>
<p>Thanks in part to MDA’s strong advocacy efforts, all 50 states and Washington, DC, implemented newborn screening for spinal muscular atrophy (SMA) in 2024, making early diagnosis and intervention possible for every newborn with this condition.</p>
<a href="https://www.votervoice.net/MDA/1/campaigns" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<h2>2024</h2>
<h3>MDA College Scholarship Program Established</h3></span>
<p>In 2024, MDA introduced a college scholarship program designed to empower young people with neuromuscular disease as they pursue higher education and career training. This program provides financial support to students, helping to alleviate barriers to educational opportunities and open pathways to personal and professional growth.</p>
<a href="https://www.mda.org/scholarship" target="_blank"><button class="noselect banner2"><span class="text">Learn More</span><span class="icon-banner2"><div class="pickgradient"><img src="../../../wp-content/uploads/2024/12/Vector.png" alt=""></div></span></button></a>
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<p>Your generosity fuels cutting-edge research, expands access to specialized care and support, and builds a strong, inclusive community for people living with a neuromuscular disease.</p>
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<p>Raise your voice to champion policies that remove barriers and broaden access. Help create a future where people with neuromuscular conditions can live with greater independence and opportunity.</p>
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