US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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What We've Achieved

MDA is the nation’s largest nonprofit supporter of research on more than 40 different neuromuscular diseases for more than 70 years. Every year MDA supports hundreds of physicians and scientists in the fight to develop effective treatments for muscular dystrophies, motor neuron diseases, and related muscle-debilitating diseases so that people living with these disorders can thrive and pursue their passions.

Since its inception, MDA has invested more than $1 billion collectively in the laboratories of 7,000 scientists, helping build the entire field of neuromuscular disease biology and pioneering technologies such as the identification of disease-causing genes, gene therapy, antisense oligonucleotides, and, most recently, gene editing.

“The basic science research that is annually supported by the MDA is like a fountain, springing forth new discoveries that drive future treatments and cures.” — Dr. Joe Metzger, University of Minnesota

This investment is bearing fruit. To date, MDA-sponsored research has led to the development of seven FDA approved therapies including Rilutek® for ALS, Myozyme® for Pompe disease, Keveyis® for periodic paralysis, Spinraza® for spinal muscular atrophy (SMA), and Exondys 51®, Emflaza®, and Vyondys 53® for Duchenne muscular dystrophy (DMD). MDA also funded the first gene therapy trial for any form of muscular dystrophy in 1999 and the first vector-based gene therapy trial for DMD in 2006. All three gene therapies in clinical testing for Duchenne in the US now are based on strategies developed with MDA funding. In 2019, MDA was awarded the Sonia Skarlatos Public Service Award by the American Society for Cell and Gene Therapy for its work in advancing gene therapy.

“In the early years few people believed that gene therapy would ever work. However, MDA believed in my ideas, and without their support, the AAV/micro-dystrophins that are currently being tested in clinical trials for DMD would have never been developed.” — Dr. Jeff Chamberlain, University of Washington

Even where no “cure” is available yet, MDA research has resulted in better treatments that result in increased survival and better quality of life.

MDA funding also facilitates the careers of bright young scientists through its Development Grants program. To ensure the long-term success of neuromuscular research, this program funds the best and brightest young researchers to become future neuromuscular disease scientists and clinicians. These grants pair exceptional mentors with promising grantees to ensure high-quality training in neuromuscular disease research. To date, MDA has helped train more than 2,000 young scientists.

“I really wouldn’t be able to do what I am doing now without the initial support from MDA.” — Dr. Jinsey Andrews, Columbia University

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