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)
Latest Information Update: 16 Feb 2024
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
- 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