Overview

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

Status:
Completed
Trial end date:
2019-08-19
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.
Phase:
Phase 1
Details
Lead Sponsor:
Sarepta Therapeutics, Inc.