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Muscular Dystrophy Association Announces More Than $2.7 Million For Research Grants to Advance Breakthroughs Across Neuromuscular Diseases
Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial myopathy, myotonic dystrophy (DM), and MDA Development Grants, which are fellowships for early career investigators.
New York, Wednesday, December 10, 2025 — The Muscular Dystrophy Association (MDA) today announced more than $2.7 million in new research grants to advance groundbreaking discoveries across multiple neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth (CMT) disease, Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), mitochondrial myopathies, and myotonic dystrophy (DM). This round of funding also includes MDA Development Grants, which are fellowships supporting early career investigators. View all MDA Research Grants here.
In addition, MDA recently awarded nearly $2 million in collaboratively funded grants, bringing its new research commitments to nearly $5 million. These investments reflect MDA’s ongoing commitment to accelerating treatments across the more than 300 neuromuscular conditions in its mission through strategic support of pioneering science and collaboration with leading investigators.
The 2025 MDA Research Grants will be awarded to the following recipients, along with additional collaborative awards highlighted here.
“Each of these new research awards exemplifies the Muscular Dystrophy Association’s mission to transform scientific discoveries into treatments for people living with neuromuscular disease,” said Sharon Hesterlee, PhD, President and CEO, Muscular Dystrophy Association. “Collaborating with researchers at top scientific institutions allows us to accelerate progress and expand the reach of therapies for the families we serve who need them most.”
Among the 2025 MDA Research Grant recipients:
- Robert Bloch, PhD, University of Maryland, will receive $300,000 for “Monitoring FSHD with Antibody to SLC34A2.” Facioscapulohumeral muscular dystrophy (FSHD), the third most common form of muscular dystrophy, is caused by the harmful, sporadic activation of the DUX4 gene in muscle fibers, leading to progressive tissue loss. This project aims to detect affected muscle fibers at the earliest stages of DUX4 activity and investigate a newly identified DUX4-activated gene that may drive muscle degeneration, with the goal of improving disease monitoring and accelerating the evaluation of emerging therapies.
- Valentina Emmanuele, MD, PhD, The Trustees of Columbia University in the City of New York, will receive $300,000 for “A pilot study of Forodesine Treatment for Patients with GUK1 Deficiency.” This project will test the potential of forodesine as a treatment for mitochondrial DNA depletion syndromes caused by GUK1 deficiency, a newly identified mitochondrial disease with severe muscular and systemic symptoms.
- Vandana Gupta, PhD, Brigham and Women’s Hospital, Inc., will receive $299,374 for “Genetic and Extrinsic Mechanisms in Metabolic Myopathies.” This project will define how genetic vulnerabilities and extrinsic stressors converge to drive metabolic dysfunction and will identify key biomarkers and therapeutic targets. The research will also evaluate both small-molecule therapies and gene-replacement strategies as potential interventions. Together, these efforts aim to accelerate treatment development and improve disease management for individuals living with metabolic myopathies.
- Chad Heatwole, MD, MS-CI, University of Rochester, will receive $297,393 for “LEOPARD DM2: Longitudinal Endpoint Optimization to Provide an Assessment of Relevant Drugs in Myotonic Dystrophy Type-2.” This research will conduct a remote study of patients with myotonic dystrophy type-2 to better understand how disease progresses in myotonic dystrophy, identify factors that are associated with slower or faster disease progression, optimize existing outcome measures, and facilitate the design of future clinical trials.
- Nicholas Maragakis, MD, Johns Hopkins University School of Medicine, will receive $299,340 for “Modeling ALS cortical neuron influences on corticospinal connectivity.” Disrupted communication between corticospinal neurons and spinal motor neurons is believed to drive disability and disease spread in ALS, yet these complex human neural connections are not accurately reproduced in current animal models. This project uses a novel hiPSC-based microfluidic system to model these networks, track real-time electrical activity, and manipulate TDP-43 to uncover how cortical pathology alters corticospinal signaling; providing a powerful platform for understanding disease mechanisms and advancing therapeutic development.
- Anirban Roy, PhD, University of Houston, will receive $300,000 for “Therapeutic Targeting of TAK1 in Duchenne Muscular Dystrophy.” Duchenne muscular dystrophy (DMD) is a severe genetic disease with limited treatment options, and our research identifies the protein TAK1 as a key driver of muscle degeneration and inflammation in DMD. This project will define the TAK1-mediated mechanisms that promote muscle pathology and evaluate small-molecule TAK1 inhibitors as potential therapeutic candidates, with the goal of identifying new treatment options.
- Charlotte Sumner, MD, Johns Hopkins University School of Medicine, will receive $300,000 for “Advancing Treatment for TRPV4 Channelopathy.” This study uses humanized mouse models to test TRPV4 blockers as potential therapies for forms of Charcot-Marie-Tooth disease and distal spinal muscular atrophy.
- Ashlee Long, PhD, Northwestern University, will receive $210,000 for “The ECM–Macrophage Axis Promotes Disease Progression in Muscular Dystrophy.” This research studies how inflammation and extracellular matrix composition contribute to Duchenne muscular dystrophy progression and evaluates potential pro-regenerative treatment strategies.
- Janani Parameswaran, PhD, Emory University, will receive $210,000 for “Investigating Skeletal Muscle-Mediated Non-Cell Autonomous Neurodegeneration.” This ALS-focused project examines how skeletal muscle contributes to neurodegeneration in C9orf72-linked disease, helping identify new therapeutic targets.
- Benjamin Cartes Saavedra, PhD, Thomas Jefferson University, will receive $210,000 for “MICU1 Loss-Related Myopathy: Role of Mitochondrial Fusion.” This project explores how MICU1 regulates mitochondrial fusion and muscle regeneration, with implications for improving understanding and treatment of mitochondrial myopathies.
Among the 2025 MDA Development Grant fellowship recipients:
These grants represent MDA’s strategic investment in both early-stage and translational research with the potential to directly impact clinical care and therapeutic development for people living with neuromuscular diseases.
“The Muscular Dystrophy Association has been at the center of neuromuscular research breakthroughs for more than seven decades,” added Angela Lek, PhD, Chief Research Officer, Muscular Dystrophy Association. “These new projects span the full research continuum—from identifying disease mechanisms to testing novel therapeutic approaches—and each one brings us closer to empowering more families to live longer more independent lives as we move forward together in the next 75 years.”
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 contact press@mdausa.org.
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.
