Trial Profile

Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7. Micro-dystrophin (microDys-IV-001)

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 07 Oct 2024

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At a glance

26 Sep 2024 According to a Sarepta Therapeutics media release, the Company will present data from the study at the 29th Annual Congress of the World Muscle Society 2024 Congress (WMS 2024), taking place Oct. 8-12, 2024, in Prague, Czechia.
01 Jan 2024 Results published in the Muscle and Nerve
22 Jun 2023 According to a Sarepta Therapeutics media release, company has received U.S.FDA accelerated approval of ELEVIDYS for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy.

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