Emma and Maddie

Clinical Trials

Clinical trials play a key role in the discovery and development of new treatments for neuromuscular diseases. The University of Florida is a major site for rare disease research working on improved therapies for on spinal muscular atrophy, Duchenne muscular dystrophy, Pompe disease, and Friedreich ataxia. As a result, our patients are among the first to benefit from new discoveries. So far these have included the first ever commercial infusions of EXONDYS 51, VYONDYS 53, and ZOLGENSMA.

If your child does not qualify for any actively enrolling trials at our site, we will assist you in finding a trial your child can participate in around the country. You are also welcome to use the tools below to find a clinical trial.

Neuromuscular Research -University of Florida

The University of Florida Powell Center for Rare Disease Research and Therapy is dedicated to improving the lives of those suffering from rare diseases by providing world-class medicine and groundbreaking research. Our center has developed an interdisciplinary research program that integrates clinical, translational and basic research strategies that allows us to deliver quality services with compassion and excellence.

MDA Clinical Trial Finder Tool

The Muscular Dystrophy Association (MDA) has a web-based Clinical Trials Finder Tool powered by Antidote. Designed to help you locate ongoing clinical trial participation opportunities across the country, the tool asks a series of questions to pinpoint trials specific to disease type, progression, age and other eligibility criteria. In just a few minutes, you can gain access to vital research that directly impacts you and families like yours. is a registry of clinical trials to use as a resource in finding. It is run by the United States National Library of Medicine at the National Institutes of Health, and is the largest clinical trials database in the world.