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Gene therapy for Sanfilippo syndrome.

1 report so far. New developments are added to this thread as they happen.

  1. Sep 17, 2026
    FDA APPROVES FAYUVI, A FAMILY-FUNDED GENE THERAPYFDA approval · 17 kids

    FDA approves first gene therapy for a rare, fatal child brain disease

    Fayuvi, a one-time infusion for children with Sanfilippo syndrome type A, won US approval after families spent years raising money to keep the early research alive.