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FDA Approves Ultragenyx's Sanfilippo Syndrome Gene Therapy
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Fusion42 · 17 September 2026 · Fusion42 review
The US FDA has approved Ultragenyx Pharmaceutical's gene therapy Fayuvi for pediatric patients with Sanfilippo syndrome type A, a rare neurodevelopmental disease.
This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 17 Sep 2026 and 28 Sep 2026.
◆ ◆ The Wire takeaway
Gene therapy founders focused on rare pediatric diseases in the US face a more open market with FDA approval creating a concrete commercial pathway. You can now plan to engage early with payers and providers for this model that just broke through regulatory barriers.
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9 sources · first reported 17 Sep 2026 · latest 28 Sep 2026
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