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A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States

Trial Profile

A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 06 May 2025

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At a glance

  • Drugs Patidistrogene bexoparvovec (Primary)
  • Indications Limb girdle muscular dystrophies
  • Focus Adverse reactions; Proof of concept
  • Acronyms DISCOVERY
  • Sponsors Sarepta Therapeutics

Most Recent Events

  • 15 Apr 2025 According to a Sarepta Therapeutics media release, enrollment and dosing is complete in this trial.
  • 15 Apr 2025 Status changed from recruiting to active, no longer recruiting.
  • 10 Feb 2025 Status changed from not yet recruiting to recruiting.

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