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

About Us

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

MDA Awards more than $12 Million in Grants


TUCSON, Ariz. — The Muscular Dystrophy Association’s latest round of grants allocates more than $12 million to the support of 38 research projects investigating the causes of, and potential treatments for, a number of forms of neuromuscular disease.

“MDA is strongly committed to funding important and exciting basic research that will serve as the basis of future therapies for neuromuscular disease,” said MDA Chairman of the Board R. Rodney Howell, M.D. Howell is a professor of pediatrics in the Miller School of Medicine at the University of Miami (Fla.).

The new grants, which became effective Feb. 1, support research into more than 15 diseases in MDA's program, including Duchenne muscular dystrophy (DMD), ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease), spinal muscular atrophy (SMA), facioscapulohumeral muscular dystrophy (FSHD), and others.

MDA Grants Set Sights on Therapies

In addition to funding investigations into underlying disease mechanisms, the grants will help guide the development of strategies for diagnosis and treatment.

For example, in Pompe disease (acid maltase deficiency), scientists will look for ways to improve the current FDA-approved treatment — enzyme replacement therapy — which sometimes leads to an unwanted immune response.

MDA funding also will help refine exciting experimental therapies such as exon skipping in DMD, and gene therapy in DMD and SMA.

“It is exciting that several of these new basic research projects are focused on further refinement of existing therapies,” noted MDA Vice President of Research Sanjay Bidichandani, M.B.B.S., Ph.D.

A number of the new grants are for projects using muscle stem cells, and other strategies to improve muscle regeneration and create new muscle fibers.

“It’s heartening to see that so much of the newly funded research builds upon recent scientific advances,” said MDA Interim President and Medical Director Valerie Cwik, M.D. “This is a clear case of great science driving great research.”

Six grants are career development grants (DGs) designed to increase the number of scientists committed to working on neuromuscular disease research.

The new grants were approved by MDA's Board of Directors based on recommendations from the Association's Scientific and Medical Advisory Committees.

For detailed information on all 38 grants, read MDA Commits $12 Million to Neuromuscular Disease Research and MDA Awards $2 Million in ALS Grants. Also see Grants at a Glance for a slideshow feature with photos and information on the new MDA grantees and their research. To review approximately 300 grants currently being funded by MDA, download this PDF.

About MDA

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.

For more information on the Association's research and other programs, visit