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Project

Clinical Trials for Muscular Dystrophy

Center:

CA-Tarjan Center UCLA, UCEDD

Fiscal Year:

2024

Contact Information:

Eileen Fowler
[email protected]
310-825-4028

Project Description:

Duchenne muscular dystrophy (DMD) and Spinal Muscular Atrophy (SMA) are genetic diseases that primarily affect males and is currently fatal. Until very recently, there have been no targeted treatments for theses disease, which affect skeletal, pulmonary and cardiac muscle. New breakthroughs in medicine have led to the development of novel therapeutics. At this time, we are participating in 10 clinical trials for DMD and SMA. 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 these currently fatal diseases.

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