An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)
Latest Information Update: 22 Aug 2024
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At a glance
- Drugs MYO 201 (Primary)
- Indications Limb girdle muscular dystrophies
- Focus Adverse reactions; Proof of concept
- Acronyms NAVIGENE
- Sponsors Sarepta Therapeutics
- 13 Nov 2023 Status changed from recruiting to active, no longer recruiting.
- 02 Aug 2023 According to a Sarepta Therapeutics media release, the first patient has been dosed in this study, in second quarter.
- 21 Dec 2017 New trial record