Condition

Sanfilippo Syndrome Type A

Treatments and trials tagged Sanfilippo Syndrome Type A, with the same country and access zoom as New Developments.

All New Developments

Sanfilippo Syndrome Type A breakthroughs

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FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Sanfilippo Syndrome Type A

Fayuvi (rebisufligene etisparvovec-hopf) — one-time IV gene therapy for children with Sanfilippo syndrome type A (MPS IIIA)

FDA approval on 17 September 2026 for Fayuvi (rebisufligene etisparvovec-hopf) suspension — a one-time AAV9 gene therapy given by intravenous…

This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved…