Trial Profile

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 11 Jun 2025

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At a glance

04 Jun 2025 According to Sarepta Therapeutics media release, company announced that the rAAVrh74 viral vector SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration.
15 Apr 2025 According to Sarepta Therapeutics media release, the company to submit a BLA to the U.S. FDA in the second half of 2025. Data expected in mid 2025.
04 Mar 2025 Planned primary completion date changed from 31 Jan 2025 to 4 Mar 2025.

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