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active, not recruiting

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

key information

study id #: NCT01539772

condition: Becker Muscular Dystrophy

status: active, not recruiting


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.

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01539772

last updated: November 22, 2018