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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)

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Trial Profile

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)

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 22 Nov 2023

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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

    • Most Recent Events

    • 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
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