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Gene Therapy
PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy
Recruiting1 awardPhase 1
Durham, North Carolina
This trial is a safety and tolerability study of a new drug for Duchenne muscular dystrophy. The drug will be given to 22 people, some of whom will also take sirolimus. The trial will be conducted in two phases, with a review by an external data monitoring committee before progressing to the next phase.
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