FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

By Sep 17, 2026

Regulatory Approvals — FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease STAT

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Auto-published by the finit.news Events monitor from Regulatory Approvals; summarized by AI and may contain errors — verify against the primary source. September 17, 2026.

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FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

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FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

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