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Long-term Follow-up Study of UX111 in MPS IIIA
NCT04360265Phase 3Enrolling by invitation
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2020
- Primary completion
- Aug 2027
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 3
01
Overview
A Phase 3 trial of rebisufligene etisparvovec in Mucopolysaccharidosis Type IIIA, sponsored by Ultragenyx Pharmaceutical Inc.. Started Sep 2020; status: enrolling by invitation.
02
Drugs
rebisufligene etisparvovec
03
Conditions
Mucopolysaccharidosis Type IIIA
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