A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg, and 30 mg/kg), administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD); To evaluate dystrophin protein level in skeletal muscle tissue following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A
