5d ago

FDA approves first-ever treatment for Sanfilippo Syndrome Type A, FAYUVI™

A gene therapy (AAV delivery of a gene encoding a specific enzyme) has been given FDA approval for treating San Filippo in children. curesanfilippofoundation.org/2026/09/fda-... FDA granted standard full approval of FAYUVI™, rebisufligene etisparvovec-hopf, the first-ever FDA-approved treatment for Sanfilippo Type A.

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FDA approves first-ever treatment for Sanfilippo Syndrome Type A, FAYUVI™

stream:bsky-jetstreamother5d ago kagi ↗

A gene therapy (AAV delivery of a gene encoding a specific enzyme) has been given FDA approval for treating San Filippo in children. curesanfilippofoundation.org/2026/09/fda-... FDA granted standard full approval of FAYUVI™, rebisufligene etisparvovec-hopf, the first-ever FDA-approved treatment for Sanfilippo Type A.