Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265 · clinicaltrials.gov ↗

PHASE3

Phase

ENROLLING_BY_INVITATION

Status

41

Enrollment

INDUSTRY

Sponsor class

Conditions

Mucopolysaccharidosis IIIA
MPS IIIA
Sanfilippo Syndrome
Sanfilippo A

Interventions

OTHER: No Investigational Product
DRUG: Adjuvant Immunomodulatory (IM) Therapy

Sponsor

Ultragenyx Pharmaceutical Inc