Description
Seeking boys aged 7 to 21 with Duchenne Muscular Dystrophy with an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment for a study of the highest tolerated dose of an investigational drug and if it increases dystrophin levels.
Overview
Participants in this study will be randomly assigned to one of two dose groups of an investigational drug. The drug will be infused once every 4 weeks. There will be about 56 study visits over 2 years.
What we're hoping for
We are studying the highest tolerated dose of an investigational drug and its effect on dystrophin levels in boys with Duchenne Muscular Dystrophy.
Additional Information
ClinicalTrials.gov Identifier: NCT04004065