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A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

Trial Profile

A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

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

Latest Information Update: 16 Feb 2024

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At a glance

  • Drugs Bidridistrogene xeboparvovec (Primary)
  • Indications Limb girdle muscular dystrophies
  • Focus Adverse reactions; First in man; Pharmacogenomic; Registrational
  • Sponsors Myonexus Therapeutics; Sarepta Therapeutics
  • Most Recent Events

    • 09 Feb 2024 Planned End Date changed from 1 Feb 2025 to 14 Feb 2027.
    • 09 Feb 2024 Planned primary completion date changed from 1 Feb 2025 to 14 Feb 2027.
    • 01 Jan 2024 Interim Post hoc exploratory analysis results assessing safety and efficacy of bidridistrogene xeboparvovec for limb-girdle muscular dystrophy published in the Nature Medicine
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