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FDA Approves First Gene Therapy for Kids With Sanfilippo Syndrome Type A

The FDA approved Ultragenyx’s Fayuvi for Sanfilippo syndrome type A following a manufacturing-related delay in 2025. Children ages 2–5 who received the treatment maintained or improved their cognitive scores.

FDA Approves First Gene Therapy for Kids With Sanfilippo Syndrome Type A
Credit: Ultragenyx
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