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LYS-SAF302 intracerebral gene therapy for MPS IIIA
NL-OMON52445Phase 3Recruiting
- Sponsor
- Blue Daisy
- Start
- Apr 2019
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 3, open-label, single-arm trial of Olenasufligene relduparvovec in mucopolysaccharidosis type III A, sponsored by Blue Daisy. Primary endpoint: Cognitive developmental quotient ratio at 24 months. Started Apr 2019; status: recruiting.
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Drugs
Olenasufligene relduparvovec
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Conditions
mucopolysaccharidosis type III A
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