Clinical Trials for Males with Duchenne Muscular Dystrophy
Project Description:
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. In this disease, there is a failure to produce dystrophin leading to a lack of integrity of the muscle cell membrane. This causes the muscle to breakdown and eventually become non-functional. Boys are typically diagnosed between 3-5 years of age. In the majority of cases, walking ability begins to decline by age 8 and the use of a wheelchair is required by age 13 years. New breakthroughs in medicine have led to the development of novel therapeutics. Exon skipping therapies result in the production of dystrophin and show great promise. Additional trials aimed at preventing muscle damage using other mechanisms have also begun. At this time, my lab is participating in eleven clinical trials for DMD. My staff consists of 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.
Keyword(s):
Research, drug, muscle, Fatal disease, Duchenne Muscular Dystrophy
Core Function(s):
Performing Research or Evaluation
Area of Emphasis
Health-Related Activities
Target Audience:
Children/Adolescents with Disabilities/SHCN
Unserved or Under-served Populations:
Specific Groups
Primary Target Audience Geographic Descriptor:
National
COVID-19 Related Data:
N/A
