MYO 103
Alternative Names: MYO103; rAAVrh74.MHCK7.hSGCG; SRP-9005Latest Information Update: 19 Jun 2025
At a glance
- Originator Nationwide Children's Hospital
- Developer Sarepta Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference; Sarcoglycan replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase III Limb girdle muscular dystrophies
Most Recent Events
- 04 Jun 2025 Phase-III clinical trials in Limb girdle muscular dystrophies (In adults, In adolescents, In children) in USA (IV) (NCT06952686)
- 01 May 2025 Sarepta Therapeutics plans a phase III COMPASS trial for Limb girdle muscular dystrophies (In children, In adults, In adolescents) in USA (IV, infusion) (NCT06952686)
- 23 Apr 2025 MYO 103 is still in preclinical development in Limb-girdle-muscular-dystrophies in USA (Parenteral)