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Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.
MDA’s Urgent Call to Action: Lift Impediments to Collaborations on Research and Care Progress
Statement from the Muscular Dystrophy Association, February 14, 2025 – Neuromuscular diseases are rare, often devastating, and it is incredibly difficult to find and deliver breakthrough potential treatments and cures.
This is why we call on the Administration to immediately lift the continuing freeze on collaborations between government and non-governmental experts and fully resume communication between health agencies and their partners.
Recent actions by the Administration have impeded collaborations between the National Institutes of Health (NIH), the Centers for Disease Control and Prevention (CDC), the Food and Drug Administration (FDA), and the Health Resources and Services Administration (HRSA) (among others) and MDA and the neuromuscular disease research and clinical community. A key NIH expert was unable to co-chair MDA’s recent summit on gene therapy, meetings have been canceled between NIH and partnering advocacy organizations, and NIH experts have dropped out of MDA’s Clinical and Scientific Conference, the largest gathering of neuromuscular experts in the world. HRSA postponed the quarterly meeting of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) in which newborn screening for Duchenne muscular dystrophy was to be discussed. FDA postponed a conversation on approaches to gene therapies and how the patient community can get involved. Numerous additional collaborations have been interrupted by these actions.
Undue delays in the research and development of diagnostic and therapeutic approaches allows neuromuscular diseases to irreversibly progress. Combined with last week’s announced 15 percent cap on NIH’s indirect research funds, impeding research collaborations on neuromuscular diseases will only further harm our community.
MDA implores the Administration to immediately lift all prohibitions on scientific and research collaboration and communication.
About Muscular Dystrophy Association
For 75 years, the Muscular Dystrophy Association (MDA) has led the way in advancing basic and translational research in treatments and cures for the neuromuscular disease community. MDA is the largest funder of research into neuromuscular diseases after the Federal government, has founded the field of genetic neuromuscular medicine, and routinely convenes the scientific and research community to accelerate scientific breakthroughs, all in service of the neuromuscular disease community.
