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AAV8 ARSB liver-directed gene therapy study in MPS VI
NL-OMON47624Phase 2Not yet recruiting
- Sponsor
- Fondazione Telethon
- Start
- May 2017
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial in mucopolysaccharidosis type VI, sponsored by Fondazione Telethon. Primary endpoints: Overall short-term and long-term safety and tolerability, Liver inflammation and 3 more. Planned start May 2017; status: not yet recruiting.
02
Conditions
mucopolysaccharidosis type VI
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