Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

NCT03300453 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

4

Enrollment

INDUSTRY

Sponsor class

Conditions

Sanfilippo Syndrome B

Interventions

DRUG: rAAV2/5-hNAGLU

Sponsor

UniQure Biopharma B.V.

Collaborators

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