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SRP-9001 open-label gene delivery study in children under 4 years with Duchenne muscular dystrophy
2023-509901-57-00Phase 2Authorised
- Sponsor
- Roche
- Start
- Oct 2023
- Modality
- Gene Therapies
- Countries
- 6
01
Overview
Phase 2, open-label trial of delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD) (2 cohorts), sponsored by Roche. Primary endpoints: Incidence of treatment-emergent adverse events, Incidence of serious adverse events and 3 more. Planned start Oct 2023; status: authorised.
02
Drugs
delandistrogene moxeparvovec
03
Conditions
Duchenne muscular dystrophy (DMD)
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