Clinical Trial Update: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome [Phase 1, Phase 2]

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Summary

The Phase 1/2 intracerebral gene therapy trial for Sanfilippo syndrome type A (Mucopolysaccharidosis type IIIA) sponsored by Lysogene has completed.

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome Type

Snippet

Sponsor: LYSOGENE | Conditions: Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A | Phase 1, Phase 2 | Status: COMPLETED