Description
Seeking boys aged 1 to less than 12 years old who have Duchenne muscular dystrophy for a study of an investigational gene therapy to cause muscle cells to produce microdystrophin.
Overview
Participants in this study will get a single dose of an investigational gene therapy. In preparation for the gene therapy participants will need therapy to suppress their immune system. There will be about 26 study visits over 26 months. Some of the study visits may take place over several days.
What we're hoping for
We are studying whether an investigational gene therapy is safe and effective in getting muscle cells to produce microdystrophin in boys aged 1 to less than 12 with Duchenne muscular dystrophy.
Additional Information
ClinicalTrials.gov Identifier: NCT05693142