Muscular Dystrophy Association Announces 70 Research Grants Totaling Over $17 Million

On Sept.19, the Muscular Dystrophy Association (MDA) announced the awarding of 70 new grants totaling over $17 million toward neuromuscular disease research. The newly funded projects aim to advance research discoveries and new therapy development in multiple areas including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy (LGMD) spinal muscular atrophy (SMA), myasthenia gravis (MG), myotonic dystrophy (DM), and many more.

Answering crucial questions to speed therapy development

This round of grant funding reinforces MDA’s unwavering commitment to the progress of neuromuscular disease research and builds on the more than $1 billion that the organization has already invested in research to uncover new treatments and cures for neuromuscular diseases since its inception. The organization’s model of funding research across many neuromuscular diseases means that findings from one disease often enable progress in others, maximizing the speed at which progress can be made. Each grant impacts neuromuscular disease research in a different way, from better understanding the underlying mechanisms of a particular disease to uncovering therapeutic targets to building clinical research infrastructure that will expedite clinical trials to providing early career support to promising young researchers.

“The Muscular Dystrophy Association has consistently been a leader in advancing treatments for individuals with neuromuscular disease. Patients are now living longer, more independent lives, due in large part to the more than $1 billion we have invested in research over the past 70+ years,” said Donald S. Wood, PhD, President and CEO of MDA. “We are pleased to invest an additional $17M during this grant cycle to continue to fund the research that will lead to increasing advancements in neuromuscular disease treatments and cures.”

“The Muscular Dystrophy Association continues to fund the most innovative research that will lead to cures for a range of neuromuscular diseases,” says Sharon Hesterlee, PhD, Chief Research Officer, MDA. “We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable.”

Highlights of the awards for the 2022 grant cycle include:

Clinical research network grants

• Jeffrey Statland (University of Kansas Medical Center), Nicholas Johnson (Virginia Commonwealth University), and Rabi Tawil and Charles Thornton (University of Rochester) were jointly awarded $1,517,502 to support their clinical trial network for FSHD, LGMD and DM. MDA will support the consolidation of project management and oversight from 15 core centers in the United States under a single umbrella of the MD Clinical Trial Research Network, aimed towards clinical trial readiness for these diseases.
• Forum Kamdar (Regents of the University of Minnesota) was awarded $198,000 towards her project to combine data from large muscular dystrophy and advanced heart failure centers in order to assess cardiac care of patients and address gaps in current cardiac care.

Research infrastructure grant

• Michael Shy (University of Iowa) was awarded $439,250 to support the Inherited Neuropathy Consortium (INC) to develop the infrastructure necessary to evaluate therapies for patients with CMT disease.

Funding for development of genetic therapies

• “Utrophin Genome Editing for Duchenne’s Muscular Dystrophy (DMD) Therapy” by Dr. Tejvir Khurana at the University of Pennsylvania
• “Developing Gene Therapy Approaches for mtDNA Deletions” by Dr. Carlo Moraes at the University of Miami
• “Linker-based gene therapy of LAMA2-related muscular dystrophy using AAVMYO” by Dr. Markus Ruegg at the University of Basel

Funding for development of stem cell-based therapies

• “Mobilizing Muscle Stem Cells to Treat DMD” by Dr. Michael Rudnicki at the Ottawa Hospital Research Institute

Funding to enhance understanding of disease mechanisms

• “Investigating a link between ER stress and nucleocytoplasmic transport in ALS” by Dr. Chang Geon Chung at the Johns Hopkins University
• Assessing the role of Fibro-Adipogenic Progenitors in EDMD” by Dr. Chiara Mozzetta at the CNT Institute of Molecular Biology and Pathology
• “Calcium regulation of ER stress and protein degradation in congenital myopathy” by Dr. Amy Hanna at the University of Queensland

Funding for diagnostic technologies

• “Long-read genome sequencing to diagnose neuromuscular disorders” by Dr. Greg Cooper at HudsonAlpha Institute for Biotechnology.

Funding for alternative therapeutic strategies to treat DMD

These projects will explore the value of repurposing FDA approved compounds and their potential benefit on DMD.

• “Mirabegron in Treating Muscle Dystrophy” by Dr. Xuhui at the University of California at San Francisco
• “Cannabidiol for the treatment of Duchenne Muscular Dystrophy” by Dr. George Rodney at the Baylor College of Medicine
• “CSF1R inhibitors as therapeutics for Duchenne Muscular Dystrophy,” by Dr. Fabio Rossi at the University of British Columbia

These are just a few of the many grants that MDA has pledged to support. For a complete list of individual awards for this grant cycle, visit MDA.org and explore the Grants at a Glance section.

About the Muscular Dystrophy Association

The Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA’s mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, Twitter, TikTok, LinkedIn and YouTube.