Gene Therapy in Patients With Mucopolysaccharidosis Disease
NCT03173521
·
clinicaltrials.gov ↗
PHASE1,PHASE2
Phase
COMPLETED
Status
9
Enrollment
OTHER
Sponsor class
Conditions
Mucopolysaccharidosis Type VI
Interventions
BIOLOGICAL:
AAV2/8.TBG.hARSB
Sponsor
Fondazione Telethon