Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

Official Title: PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.

NCT01539772
Kuntz, Nancy L., MD
Observational
No

Contact Information:

Nancy Kuntz, MD

312.227.3560