Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

NCT02053064 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

4

Enrollment

INDUSTRY

Sponsor class

Conditions

Mucopolysaccharidosis Type III A
Sanfilippo Disease Type A

Interventions

GENETIC: SAF-301

Sponsor

LYSOGENE