Clinical Trial Update: Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA [Phase 3]

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Summary

Phase 3 follow-up study of AAV-mediated gene transfer (UX111, formerly ABO-102) for Mucopolysaccharidosis type IIIA is currently enrolling by invitation.

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome Type

Drugs mentioned

• UX111

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Mucopolysaccharidosis IIIA, MPS IIIA | Phase 3 | Status: ENROLLING_BY_INVITATION