FDA approves rebisufligene etisparvovec, first gene therapy for Sanfilippo syndrome type A

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy, as the first treatment for pediatric patients with Sanfilippo syndrome type A (MPS IIIA), a rare fatal neurodegenerative disease that previously had no therapy to alter its course.

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