Clinical Trial Update: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy [Phase 1, Phase 2]

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Summary

This update reports long-term follow-up results from a Phase 1/2 trial of SAF-301 gene therapy administered intracerebrally in patients with Sanfilippo syndrome…

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome Type

Drugs mentioned

• SAF-301

Snippet

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