Clinical Trials for Muscular Dystrophy

2017

Duchenne muscular dystrophy is a genetic disease that primarily affects males and is currently fatal. Until very recently, there have been no targeted treatments for this disease, which affects skeletal, pulmonary and cardiac muscle. New breakthroughs in medicine have led to the development of novel therapeutics. At this time, we are participating in eleven clinical trials for DMD. The staff are highly trained research physical therapists and an engineer to collect outcome data for these clinical trials. Measures include muscle strength, physical functioning, pulmonary function, walking ability and health related quality of life. We are hopeful that these clinical trials will result in FDA approval of medications to slow or even cure this currently fatal disease.

Research, drug, muscle, Fatal disease, Duchenne Muscular Dystrophy

Performing Research or Evaluation

Health-Related Activities

Children/Adolescents with Disabilities/SHCN

Specific Groups

National

N/A