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

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Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

MDA Awards $6.7 Million in New Research Grants

Investment focuses on supporting the world’s best scientists and projects to find breakthroughs across diseases

CHICAGO, September 1, 2016 – The Muscular Dystrophy Association today announced the award of 25 new research, development and research infrastructure grants totaling nearly $7 million and targeted to make an impact on muscular dystrophy, ALS and related life-threatening diseases in its program. The new grants are in addition to 41 grants announced earlier this year and bring MDA’s total 2016 commitment to new research aimed at finding treatments and cures across diseases to more than $17 million.

“With this new round of grants, MDA continues to lead the fight against neuromuscular disease by funding top scientists and fueling cutting-edge research around the world aimed at accelerating treatments and cures for MDA families,” said MDA’s Executive Vice President and Chief Medical & Scientific Officer Valerie A. Cwik, M.D. “Every grant MDA funds has the potential to be the catalyst that will drive the next life-changing medical and scientific advances for every child and adult fighting neuromuscular disease.”

For more than six decades, MDA has funded basic, translational and clinical research. The power of MDA’s research program lies in its big-picture approach that leverages insights and information resulting from research in one disease area to inform and advance discoveries and breakthroughs in others. This year, MDA has reviewed 443 grant applications and awarded grants to 66 leading scientists conducting innovative research targeted to make an impact across all the diseases in MDA’s program.

The latest round of research grants was approved by MDA’s Board of Directors after careful deliberations and analysis by MDA’s Research Advisory Committee, through which leading clinicians and scientists in volunteer roles oversee the peer-review process. This year, MDA is funding 150 different research projects in 11 countries. 
Vital research and services dollars come from generous MDA partners and supporters who organize, and donate to, community fundraising programs such as this summer’s Fill The Boot drives led by the International Association of Fire Fighters; retailers who do in-store promotions such as Lowe’s, CITGO Petroleum Corp. and Casey’s General Stores; and Harley-Davidson Motor Company enthusiasts who raise funds through dealer programs, rides and special events. 

“MDA is committed to helping make therapy options available to treat all the diseases under our umbrella as quickly as possible. Thanks to generous partners and donors who champion our work and our cause, we are able to fund the research that will make that day a reality,” Cwik added. “We’re laser-focused on helping MDA families live longer and grow stronger.”  

Among the new MDA research grants:

  • Testing a treatment strategy for ALS (amyotrophic lateral sclerosis): Scientists at the University of Florida in Gainesville will work to develop an antibody therapy as a potential treatment in this devastating disease.  
  • Increasing understanding of Duchenne muscular dystrophy (DMD): Researchers at Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Calif., will work to increase understanding about the complexity of cellular interactions that underlie disease progression in DMD.
  • Shedding light on molecular mechanisms in facioscapulohumeral muscular dystrophy (FSHD): Scientists at the University of Washington in Seattle will work to increase understanding of the role of DUX4 protein in FSHD. 
  • Improving the capability to provide definitive diagnosis in limb-girdle muscular dystrophy (LGMD): Researchers at Emory University will continue groundbreaking work to identify and characterize gene defects that can cause LGMD. 
  • Enhancing the effectiveness of experimental therapies for spinal muscular atrophy (SMA): Scientists at the University of Edinburgh, Scotland, will investigate how the mechanisms underlying SMA influence the way therapies work at different stages of the disease.

Summer 2016 Grants Materials 

MDA has funded more than $1 billion in neuromuscular disease research since 1950, with developments in recent months providing proof points illustrating the strength and determination behind that commitment:

  • Encouraging results in a clinical trial to test nusinersen in infants with type 1 spinal muscular atrophy (SMA) caused the trial to be stopped to allow the participants to transition into a trial in which all can receive the drug. An expanded access program is expected to open in the coming months to make nusinersen available to all eligible babies with infantile-onset SMA. MDA funded foundational work in SMA and invested nearly $750,000 in the early-stage development of nusinersen.
  • An MDA-supported worldwide study validated surgery as a treatment for myasthenia gravis (MG). Results from the study will help to inform clinicians and patients about the potential benefits of surgery for MG and lead to improvements in care for people with the disease.
  • An FDA Advisory Committee reviewed eteplirsen to treat Duchenne muscular dystrophy (DMD). The agency’s decision is still pending; an accelerated approval could make eteplirsen the first disease-modifying drug approved by the FDA for the treatment of DMD. MDA supported the early development of eteplirsen and exon skipping via funding to Steve Wilton, then at the University of Western Australia in Perth. While it is being developed for DMD, exon skipping drugs may have application to other genetic diseases down the line.
  • The FDA accepted a New Drug Application for deflazacort to treat DMD and granted Priority Review. MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including deflazacort, in DMD, with studies to determine the drug’s mechanism of action, drug effects, side effects and best dosing regimen.

About the Muscular Dystrophy Association

MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at