Progress Through Partnership: MDA’s Collaborative Research Grants Drive Neuromuscular Science Forward

Progress in neuromuscular research has always depended on collaboration — scientists, families, advocates, and organizations uniting to accelerate the path to treatments. This spirit is at the heart of the Muscular Dystrophy Association’s latest announcement: nearly $2 million in new collaborative research grants awarded with seven partner organizations to advance breakthroughs across ALS, congenital myopathies, limb-girdle muscular dystrophy, Friedreich’s ataxia, inclusion body myositis, and mitochondrial disease.

As MDA celebrates its 75th anniversary year, these grants reaffirm a core belief: when organizations work together, progress becomes possible. Sharon Hesterlee, PhD, President and CEO at MDA, underscores this mission: “As an umbrella organization, MDA represents families living with any of over 300 neuromuscular diseases. 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.”

Advancing science together

Below is a look at each of the new research projects and the partner organizations helping to drive them forward.

Targeting Calcium Pathways in LGMD R1/2A

Partner: Coalition to Cure Calpain 3
Investigator: Elisabeth Barton, PhD, University of Florida
Project: SOCE modulation as a potential therapeutic for LGMD R1/2A

This study examines how store-operated calcium entry (SOCE) affects muscle health in LGMD2A/R1 and explores whether modulating this pathway could serve as a therapeutic strategy. Jennifer Levy, Scientific Director of Coalition to Cure Calpain 3, shared, “This collaboration with Muscular Dystrophy Association will enable us to accelerate progress and bring hope to the LGMD2A/R1 community.”

Advancing Diagnostics and Gene Therapies for Congenital Myopathies

Partners: Cure ADSSL1, Cure CMD
Investigator: Alan Beggs, PhD, Boston Children’s Hospital
Project: Molecular Genetics and Therapies for Congenital Myopathies

This project uses next-generation genomic tools and AAV-based gene therapies to uncover disease mechanisms and develop treatments for SELENON- and ADSSL1-related congenital myopathies. Priyanka Kakkar, President of Cure ADSSL1, emphasized, “For patient-founded organizations like ours, collaboration is the most powerful accelerator.” Rachel Alvarez, Executive Director of Cure CMD, added, “Collaboration is critical to advancing rare neuromuscular disease research toward treatments.”

Studying Sensory Dysfunction in Friedreich’s Ataxia

Partner: Friedreich’s Ataxia Research Alliance (FARA)
Investigator: Jordi Magrané, PhD, Weill Cornell Medicine
Project: Somatosensory dysfunction in a Friedreich’s ataxia mouse model

By analyzing early nerve and sensory changes, this project seeks to identify new therapeutic approaches for Friedreich’s ataxia. FARA’s Director of Research, Liz Soragni, PhD, shared, “Collaborating with MDA amplifies the reach and impact of this work, bringing us closer to effective treatments for FA.”

Restoring Key Cellular Pathways in ALS

Partner: ALS Network
Investigator: Matthew Nolan, PhD, Massachusetts General Hospital
Project: Developing regulators of Stathmin-2 in ALS

This project focuses on restoring Stathmin-2 — a crucial protein lost in ALS — using chemical and genetic approaches. ALS Network CEO Sheri Strahl, MPH, MBA, noted, “Hope grows stronger when it’s matched with action — and that’s exactly what this partnership delivers.”

Characterizing Immune Dysfunction in Inclusion Body Myositis

Partner: The Myositis Association (TMA)
Investigator: Bhaskar Roy, PhD, Yale University
Project: In-depth characterization of immune dysfunction in IBM

Using single-cell analysis, this study explores how immune cells contribute to muscle degeneration in IBM, aiming to identify new therapeutic targets.

Paula Eichenbrenner, MBA, CAE, Executive Director of TMA, said, “This project reflects the TMA×MDA partnership in our mutual and relentless pursuit to investigate inclusion body myositis.”

Reducing Lipotoxicity in Mitochondrial Myopathy

Partner: United Mitochondrial Disease Foundation (UMDF)
Investigator: Mariena D’Aurelio, PhD, Weill Cornell Medicine
Project: Modulating ER stress-induced lipotoxicity in mitochondrial myopathy

This study investigates how lipid buildup drives muscle dysfunction and tests therapeutic strategies that may restore metabolic health.

UMDF’s Philip E. Yeske, PhD, shared, “Collaboration is at the heart of everything we do at UMDF, so working alongside MDA aligns perfectly with our mission.”

Understanding Neuronal Changes in CoQ10 Deficiency

Partner: United Mitochondrial Disease Foundation (UMDF)
Investigator: Alba Pesini Martin, PhD, Columbia University Irving Medical Center
Project: Investigating neuronal function in CoQ10 deficiency

This research examines how lipid and metabolic abnormalities in CoQ10 deficiency affect neuronal structure, survival, and function. By uncovering how these disruptions contribute to neurological symptoms — and why current CoQ10 supplements cannot effectively reach the brain — the project aims to identify new metabolic targets for therapy. UMDF’s Science & Alliance Officer Philip E. Yeske, PhD, emphasized the power of this partnership, which also supports the mitochondrial myopathy project. “Collaboration is at the heart of everything we do at UMDF, so working alongside Muscular Dystrophy Association to co-fund mitochondrial disease research aligns perfectly with our mission,” he shared.

Building a stronger research ecosystem

Across these seven projects, one theme is clear: collaboration multiplies impact. Each co-funding partnership broadens scientific reach, strengthens community connections, and accelerates paths to treatments across multiple neuromuscular diseases.

Angela Lek, PhD, Chief Research Officer at MDA, captures this shared purpose: “Partnering with organizations that share our mission allows us to maximize the impact of every dollar we invest. Together, we are building a stronger, more connected research ecosystem for the neuromuscular disease community.”

This collaborative model has guided MDA’s research legacy for 75 years, fueling more than $1.1 billion in research investment, over 7,000 supported investigators, and more than 25 FDA-approved treatments in the last decade. Today’s newly awarded grants carry that momentum forward — honoring MDA’s history while accelerating toward a future of more breakthroughs and more hope.

Together, we advance science. Together, we strengthen our community. And together, we move closer to a world where every neuromuscular disease has meaningful treatments and pathways to care.