Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon Skipping
About: 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.
Ages Eligible for Study: 4 Years and older (Child, Adult, Senior)
Sexes Eligible for Study: Male
Status: This study is ongoing, but not recruiting participants.
For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Jessica Ehrbar, Clinical Research Coordinator.