A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD)

  • STATUS
    Recruiting
  • End date
    Mar 16, 2026
  • participants needed
    20
  • sponsor
    Sarepta Therapeutics, Inc.
Updated on 27 June 2021

Summary

This is an open-label gene delivery study evaluating the safety of and expression from SRP-9001 in individuals with Duchenne Muscular Dystrophy over 260 weeks. The primary endpoint is the change in quantity of micro-dystrophin protein expression from baseline to Week 12.

Details
Condition DUCHENNE MUSCULAR DYSTROPHY
Treatment SRP-9001
Clinical Study IdentifierNCT04626674
SponsorSarepta Therapeutics, Inc.
Last Modified on27 June 2021

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