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

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MDA Awards $14.1 Million in New Grants to Advance Treatments for Muscle and Nerve Diseases

TUCSON, Ariz. — The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

“Thanks to generous public support of MDA, we’re able to keep funding vital muscle disease research at a time when such investments by government agencies, nonprofit organizations and private sources have tightened due to severe economic pressures,” said R. Rodney Howell, M.D., chairman of the MDA Board of Directors.

During its July meeting, the MDA Board of Directors unanimously approved new funding for 38 research leaders in 20 states, Australia, Belgium, Canada, Israel, The Netherlands and the United Kingdom, along with nearly $600,000 in underwriting support for two collaborative research initiatives to speed therapeutic inroads for two of the more common neuromuscular diseases (Charcot-Marie-Tooth disease and Friedreich’s ataxia).

Ten of the investigations, representing a $3.4 million investment, focus on ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease), and may yield additional insights valuable to treating a variety of neurodegenerative diseases, including Alzheimer’s, Parkinson’s and Huntington’s diseases, spinal muscular atrophy (SMA), as well as fragile X, a genetic developmental disorder. MDA is the world’s leading nongovernmental funder of ALS research.

More than $6.9 million in new MDA funding will advance important muscular dystrophy research initiatives, including a $5.3 million infusion primarily for work that could lead to additional clinical trials of potential therapeutics for Duchenne and Becker muscular dystrophies. One such grant is for a two-year project at Children’s Hospital Boston that will test 4,000 FDA-approved compounds in zebrafish and mouse models to determine if some secondary uses for approved drugs could be fast-tracked as new treatment options for muscular dystrophy.

Another initiative, at the University of Western Australia, hopes to translate early successes in using designer molecules, called antisense oligomers, to spur the flawed gene responsible for both Duchenne and Becker dystrophies to express dystrophin, the protein that is absent or lacking in these diseases. According to Steve Wilton, Ph.D., the University of Western Australia investigator who has pioneered this therapeutic strategy with MDA funding, “the same qualities that make dystrophin expression a difficult target for gene therapy make it the best candidate for therapeutic success using genetic band aids (designer molecules).”

Here are MDA funding highlights.

UC Irvine = $372K
Stanford University = $200K
UC San Diego = $330K
UC San Diego 2 = $330K

University of Colorado = $303K

University of Florida = $179K
University of Miami = $364K

Emory University (MB) = $525K
Emory University (RW) = $359K

Loyola University = $405K

University of Louisville = $360K

Beth Israel Deaconess = $352K
UMass Medical School = $330K
Children’s Hospital = $375K

Johns Hopkins University = $348K

University of Missouri = $160K

University of Arizona = $375K

University of New Mexico = $340K

New York City
Columbia University (SY) = $180K
Columbia University (HO) = $375K
Columbia University (WH) = $311K

Thomas Jefferson University = $180K

St. Jude’s Children’s Hospital = $180K

Methodist Neuro. Institute = $330K
University Texas, Houston = $302K
Baylor College of Medicine = $411K

Salt Lake City
Sfida Biologic, Inc. = $79K

University of Washington = $313K

University of Melbourne = $375K
Univ. of Western Australia = $368K

University of Ottawa = $180K
University of Ottawa B = $360K
Quebec, Université Laval = $345K

Tel-Aviv University = $360K

Research grant applications are peer-reviewed twice yearly by MDA’s Medical and Scientific Advisory Committees, comprising world-renowned experts in neuromuscular disease research. The most promising of some 500 applications received each year are recommended for funding to the MDA Board of Directors.

The importance of MDA funding is aptly described by Dawn Cornelison, Ph.D., at the University of Missouri in Columbia: “MDA is a lifeline for a lot of scientists, as well as patients and their families.”

Her sentiments are shared by other MDA researchers, including Kurt Beam, M.D., who said, “The funding from MDA is very important because National Institutes of Health funding has become increasingly uncertain and tends to be focused on areas such as cancer, cardiovascular disease and mental disease.”

Often credited for its leadership in building the field of neuromuscular disease research, MDA offers enhanced clinical care for individuals affected by muscle disorders, achieving important quality of life and longevity gains. The Association, which serves the more than 1 million Americans affected by neuromuscular diseases, is the first nonprofit to earn a Lifetime Achievement Award from the American Medical Association (“for significant and lasting contributions to the health and welfare of humanity”).

MDA-funded scientists have uncovered the genetic defects that cause several forms of muscular dystrophy; Charcot-Marie-Tooth disease (CMT); a form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease); childhood spinal muscular atrophy (SMA) and other neuromuscular conditions. Now entering a period of increasing numbers of clinical trials of potential therapeutics, the Association’s network of approximately 200 hospital-affiliated clinics is instrumental in identifying appropriate candidates for clinical trials; and to help refine outcome measures for those clinical trials.

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 MDA research and programs, go to

For more information about these new grants, visit MDA's "Grants at a Glance," an online slideshow that showcases each grant with photos and detailed information.