Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7. Micro-dystrophin (microDys-IV-001)
Latest Information Update: 12 Jan 2024
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
- 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.