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Phase I/II AAV9-hSGSH Gene Therapy in MPSIIIA
2024-515835-31-00Phase 2Completed
- Sponsor
- ESTEVE Pharmaceuticals
- Start
- Jul 2024
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of AAV9-hSGSH in mucopolysaccharidosis type III A, sponsored by ESTEVE Pharmaceuticals. Primary endpoints: Physical examination, Adverse events and 6 more. Started Jul 2024; status: completed.
02
Drugs
AAV9-hSGSH
03
Conditions
mucopolysaccharidosis type III A
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