Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

PHASE1,PHASE2

Phase

TERMINATED

Status

Enrollment

INDUSTRY

Sponsor class

Stopped Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing

Conditions

MPS IIIA
Sanfilippo Syndrome
Sanfilippo A
Mucopolysaccharidosis III

Interventions

DRUG: ABO-102

Sponsor

Ultragenyx Pharmaceutical Inc

Collaborators

[object Object]