Clinical Trial Update: Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome [Phase 1, Phase 2]

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Summary

A phase 1/2 clinical trial investigating intracerebral gene therapy for Sanfilippo syndrome type B has completed.

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Diseases / indications

• Sanfilippo Syndrome Type

Snippet

Sponsor: UniQure Biopharma B.V. | Conditions: Sanfilippo Syndrome B | Phase 1, Phase 2 | Status: COMPLETED