Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
Status:
Completed
Trial end date:
2019-11-27
Target enrollment:
Participant gender:
Summary
This is an open-label, phase I/II study of intra-cerebral administration of
adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from
Sanfilippo type B syndrome.