FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A - Poll - MDSpire

FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

Fayuvi is authorized for pediatric patients with preserved neurodevelopmental function following a study comparing treated children with a natural history cohort

September 30, 2026
3 min

The Medicine Maker

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The Medicine Maker

FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

September 30, 2026

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