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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: 27 May 2024

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At a glance

Drugs Delandistrogene moxeparvovec (Primary)
Indications Duchenne muscular dystrophy
Focus Adverse reactions; Proof of concept; Registrational
Acronyms microDys-IV-001
Sponsors Sarepta Therapeutics

Most Recent Events

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.
09 May 2023 Status changed from active, no longer recruiting to completed.

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