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Long-term follow-up of ABO-102 gene therapy in MPS IIIA
2023-510392-66-00Phase 3Authorised
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Nov 2020
- Modality
- Gene Therapies
- Countries
- 3
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Overview
Phase 3, open-label, single-arm trial of rebisufligene etisparvovec in mucopolysaccharidosis type III A, sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: Incidence of treatment-emergent adverse events and treatment-emergent serious adverse events and Bayley Scales of Infant and Toddler Development, Third Edition Cognitive raw score. Planned start Nov 2020; status: authorised.
02
Drugs
rebisufligene etisparvovec
03
Conditions
mucopolysaccharidosis type III A
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