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

key information

study id #: NCT03375255

condition: Muscular Dystrophy, Duchenne

status: completed


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.

intervention: SRP-5051

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03375255

last updated: April 08, 2020