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ASP2957 in ventilator-dependent pediatric X-linked myotubular myopathy (Phase 1/2)
2025-523213-29-00Phase 2Authorised
- Sponsor
- Astellas Pharma
- Start
- Feb 2026
- Modality
- Gene Therapies
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of ASP2957 in myotubular myopathy (MTM), sponsored by Astellas Pharma. Primary endpoints: Incidence and severity of TEAEs and AESIs and Change from baseline in clinical safety assessments. Planned start Feb 2026; status: authorised.
02
Drugs
ASP2957
03
Conditions
myotubular myopathy (MTM)
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