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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

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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: 26 Nov 2025

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At a glance

  • Drugs Bidridistrogene xeboparvovec (Primary) ; Glucocorticoids; Prednisone
  • Indications Limb girdle muscular dystrophies
  • Focus Registrational; Therapeutic Use
  • Acronyms EMERGENE
  • Sponsors Sarepta Therapeutics

Most Recent Events

  • 03 Oct 2025 Sarepta will present results from the EMERGENE phase 3 study on the expression of SGCB and safety following bidridistrogene xeboparvovec treatment in patients with LGMD2E/R4, at the 30th Annual Congress of the World Muscle Society (WMS) 2025 Congress, taking place Oct. 7-11, 2025, in Vienna, Austria
  • 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.

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