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Muscular Dystrophy Association Announces Research Grants Totaling $8.4 Million

NEW YORK, N.Y., September 13, 2023 — The Muscular Dystrophy Association (MDA) announced today the awarding of 37 new grants with a total funding commitment of $8.4 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), Emery-Dreifuss muscular dystrophy (EDMD), limb-girdle muscular dystrophy (LGMD), spinal muscular atrophy (SMA), myotonic dystrophy (DM), and many more.

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 the organization has already invested in research to uncover new disease-modifying treatments for neuromuscular diseases since its inception. The organization’s model of funding research across many neuromuscular diseases means findings from one disease often enables 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 people living with muscular dystrophy, ALS and related neuromuscular diseases,” said Donald S. Wood, Ph.D., President and CEO, Muscular Dystrophy Association. “This organization has built the field of neuromuscular disease research with innovative and strategic grant funding that has now led to the first ever gene therapy treatments for Duchenne muscular dystrophy and spinal muscular atrophy. Since 2015 there have been more than 20 FDA approved treatments and people 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 73 years. We are pleased to invest an additional $8.4 million during this grant cycle to continue to accelerate advancements in treatments to empower the families we serve.”

“MDA is proud to support the most innovative research that will lead to novel treatments for a range of neuromuscular diseases,” says Sharon Hesterlee, Ph.D., Chief Research Officer, Muscular Dystrophy Association. “We are thrilled to announce 37 new grants totaling $8.4 million to further advance our grantees’ research, including new development trainee awards for the next generation of scientists.”

MDA 2023 Research Grant Awards

The following are highlights from the 2023 MDA Research Grant Awards:

• Dr. Umrao Monani Columbia (Columbia University Medical Center) was awarded a research grant in the amount of $299,999.25 to study the mechanisms and treatment of muscle pathology in SMA. This proposal investigates the role of survival motor neuron (SMN) protein in stem cells. If successful, the project will cast important light on the role of muscle in SMA and how SMN protein functions in this tissue to maintain its health.
• Dr. Lori Wallrath (University of Iowa) was awarded $296,044 to study the contributions of genetic variations to LMNA-associated muscular dystrophy. This project will test modifier genes using fruit fly models. The identification of these modifier genes will aid in the diagnosis, prognosis, and treatment of individuals with LMNA-muscular dystrophy.
• Dr. Kate Eichinger (University of Rochester) received the research grant of $299,525 for her project entitled Mobile App to Overcome Barriers and Increase Level of Exercise in Myotonic Dystrophy I (MOBILE). This project will utilize a mobile app to deliver individualized educational and behavioral strategies to promote health behavior change and increase physical activity and exercise in individuals with DM1.
• Dr. Daniel Michele (The Regents of the University of Michigan) was awarded $299,525 to study metabolic therapies for dystrophic cardiomyopathy. The goal of this project is to test metabolic modulator drugs under investigation for other diseases that could be repurposed for improving cardiac function, as well as skeletal muscle function, in DMD patients.
• Dr. Eran Perlson (Tel Aviv University) was awarded the research grant of $299,000 for his study entitled A Novel RNA Treatment for ALS. The study will evaluate the specific effects of miR126 on ALS-related gene expression. If successful, the project will not only deepen our understanding of ALS but will exceedingly facilitate the development of RNA-based therapies for ALS and related conditions.

MDA 2023 Young Investigators (Development Grantees)

The following are highlights from the MDA 2023 Development Trainee Awards:

• Dr. Andrea Armani (University of Zurich) was awarded $210,000 for his project titled Dissecting Lysosomal Signals to Fight Pompe Disease. This project proposes to set up a ground-breaking methodology for unraveling hidden signals at the onset of Pompe pathogenesis, with the expectation to find novel potential targets exploitable for boosting the current traditional enzyme replacing therapy.
• Dr. Andreas Patsalos (Johns Hopkins University School of Medicine) was awarded $210,000 to complete his study entitled Targeting Layered Regenerative Inflammation Tissue Zones in Dystrophic Muscle. This project aims to evaluate the impact of various anti-inflammatory treatments on the formation of regenerative tissue zones found to be sensitive to glucocorticoid treatment and will reevaluate the impact of current DMD therapies on muscle regeneration using state-of-art approaches. If successful, the project has the potential to introduce new combination therapies for muscular diseases to support the growth of muscle tissue.

MDA Summer Undergraduate Research Experience (SURE) Internship Awards

New this year, MDA also awarded summer research internships to undergraduates from under-represented backgrounds with the goal of increasing diversity in the field of neuromuscular research. The pilot program supported four students in undertaking research projects with volunteer mentors from MDA-supported research institutes.

Internship recipients and mentors included:

• Malaikah Wakadilo (Dr. Clotilde Lagier-Tourenne, Massachusetts General Hospital)
• Zoe Lee (Dr. Noah Weisleder, The Ohio State University’s Davis Heart and Lung Research Institute)
• Rhheaa Mehta (Dr. Udai Pandey, UPMC Children’s Hospital)
• Sasha Saxon (Dr. Matthew Alexander, University of Alabama at Birmingham)

These are just a few of the many grants the Muscular Dystrophy Association 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 Muscular Dystrophy Association

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, X, Threads, TikTok, LinkedIn, and YouTube.