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ATA-100 first-in-human phase 1/2 study in FKRP-related limb-girdle muscular dystrophy
2023-506677-36-00Phase 2Authorised
- Sponsor
- Atamyo Therapeutics
- Start
- Jul 2022
- Modality
- Gene Therapies
- Countries
- 2
01
Overview
Phase 2, open-label, single-arm trial of AAV gene therapy for LGMD 2I/R9 in limb-girdle muscular dystrophy type 2I (LGMD2I), sponsored by Atamyo Therapeutics. Primary endpoint: Safety and tolerability. Planned start Jul 2022; status: authorised.
02
Drugs
AAV gene therapy for LGMD 2I/R9
03
Conditions
limb-girdle muscular dystrophy type 2I (LGMD2I)
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