CompletedPHASE1, PHASE2NCT03300453

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Studying Mucopolysaccharidosis type 3

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Key facts

Sponsor
UniQure Biopharma B.V.
Principal Investigator
Kumaran Deiva, MD
Hopitaux Universitaires Paris-Sud
Intervention
rAAV2/5-hNAGLU(drug)
Enrollment
4 enrolled
Eligibility
All sexes
Timeline
20132019

Study locations (1)

Collaborators

Venn Life Sciences · Institut Pasteur

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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