Clinical Trial Update: Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease [Phase 1, Phase 2]

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Summary

A phase 1/2 clinical trial of ABO-102 for MPS IIIA (Sanfilippo syndrome) was terminated; results have been posted.

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome

Drugs mentioned

• ABO-102 (unlisted)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: MPS IIIA, Sanfilippo Syndrome | Phase 1, Phase 2 | Status: TERMINATED | Results posted: 2023-07-25