A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

6

Enrollment

INDUSTRY

Sponsor class

Conditions

Sanfilippo Syndrome Type A (MPS IIIA)

Interventions

DRUG: SOBI003

Sponsor

Swedish Orphan Biovitrum