New York, Dec. 03, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced the awarding of seven new collaborative research grants totaling nearly $2 million to advance understanding and treatment of multiple neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), congenital myopathies, Friedreich’s ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy (LGMD), and mitochondrial disease. Each of these grants represents a collaborative effort between MDA and partner organizations working together to accelerate the development of promising therapies. Learn more about MDA’s research grants, here.
“These new awards highlight how collaboration across organizations can speed progress and expand our reach,” said Sharon Hesterlee, PhD, President and Chief Executive Officer, Muscular Dystrophy Association. “As an umbrella organization, the Muscular Dystrophy Association represents families living with any of over 300 neuromuscular diseases — many with their own types and subtypes — so we are uniquely positioned to unite efforts across the field. By combining resources and expertise with our colleagues at disease-specific organizations, we can fund more innovative projects and accelerate the development of treatments that have the greatest potential to change lives.”
Among the newly awarded grants are:
- Elisabeth Barton, PhD, University of Florida, will receive $293,911 for her project “SOCE modulation as a potential therapeutic for LGMD R1/2A.” The study will explore the role of store-operated calcium entry (SOCE) in muscle health and its potential as a treatment target for limb-girdle muscular dystrophy type 2A/R1 (LGMD 2A/R1). This research is co-funded with Coalition to Cure Calpain 3.
- Alan Beggs, PhD, Boston Children’s Hospital, will receive $300,000 for “Molecular Genetics and Therapies for Congenital Myopathies.” Co-funded with Cure ADSSL1 and Cure CMD, this project aims to advance the diagnosis and treatment of congenital myopathies by combining next-generation genomic technologies to uncover novel disease genes and developing AAV-based gene replacement therapies for SELENON- and ADSS1-related myopathies.
- Jordi Magrané, PhD, Weill Medical College of Cornell University, will receive $300,000 for “Somatosensory dysfunction in a Friedreich’s ataxia mouse model.” This project, co-funded with Friedreich’s Ataxia Research Alliance (FARA), will explore how nerve and sensory deficits contribute to disease progression, and open new therapeutic avenues.
- Matthew Nolan, PhD, Massachusetts General Hospital, will receive $210,000 for “Developing novel chemical and genetic regulators of Stathmin-2 in ALS.” The project aims to restore key cellular pathways involved in amyotrophic lateral sclerosis (ALS) and is co-funded with the ALS Network.
- Bhaskar Roy, PhD, Yale University, will receive $299,992 for “In-depth characterization of immune dysfunction in inclusion body myositis.” The project aims to investigate how immune cells contribute to muscle degeneration in inclusion body myositis (IBM). Using single-cell analysis to study B and T cell interactions in muscle and blood, the research seeks to identify new therapeutic targets and better understand disease mechanisms. This project is to be co-funded with The Myositis Association (TMA).
- Mariena D’Aurelio, PhD, Weill Medical College of Cornell University, will receive $299,973 for “Modulating ER stress-induced lipotoxicity to ameliorate mitochondrial myopathy.” This project, co-funded with the United Mitochondrial Disease Foundation, will investigate how toxic lipid buildup and resulting cellular stress drive muscle dysfunction in mitochondrial myopathy. By testing two therapeutic strategies aimed at reducing lipotoxicity in a mouse model, the study seeks to establish a promising new metabolic target for treatment.
- Alba Pesini Martin, PhD, Columbia University Irving Medical Center, will receive $210,000 for “Investigating MAM domains and neuronal function in Coenzyme Q10 deficiency.” Co-funded with the United Mitochondrial Disease Foundation, this project will examine how lipid abnormalities linked to CoQ10 deficiency affect neuronal structure and survival. By uncovering how these changes drive neurological symptoms—and identifying potential metabolic targets—the study aims to lay the groundwork for new therapies that can overcome current challenges, including the inability of CoQ10 supplements to reach the brain.
“Partnering with organizations that share our mission allows us to maximize the impact of every dollar we invest,” said Angela Lek, Chief Research Officer, Muscular Dystrophy Association. “Together, we are building a stronger, more connected research ecosystem for the neuromuscular disease community.”
These collaborations underscore MDA’s ongoing commitment to fostering partnerships that accelerate translational research and bring treatments closer to families living with neuromuscular diseases.
About MDA Research Program
For 75 years, the Muscular Dystrophy Association (MDA) has been the nation’s leading nonprofit driving research and innovation in neuromuscular diseases—investing more than $1.1 billion to date. This commitment has fueled discoveries that have led to over 25 FDA-approved treatments in the past decade alone, transforming care and improving quality of life for individuals and families affected by neuromuscular diseases. MDA’s enduring investment has also fostered the next generation of scientific leaders—supporting more than 7,000 investigators and training over 2,000 early-career researchers whose work continues to advance new breakthroughs and bring hope and strength to the neuromuscular community.
Media inquiries contact press@mdausa.org.
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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 over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. 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, Bluesky, TikTok, LinkedIn, and YouTube.
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years of legacy, impact, and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. Learn more at MDA75.org.
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Mary Fiance, National Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org
