What is Fayuvi, the first FDA-approved treatment for Sanfilippo syndrome type A?

Fayuvi is a one-time IV gene therapy approved for eligible pediatric patients with preserved neurodevelopmental function.

1. A Qualified Treatment Center confirms diagnosis, disease stage, and whether the child fits the FDA-approved population.
2. The AAV9 vector delivers a working copy of the SGSH gene so cells can make the missing enzyme.
3. Corticosteroids are used around the infusion, with continued monitoring for liver and blood-related side effects.
4. Families should review the pivotal trial’s 23.5-point cognitive difference, nearly eight years of follow-up, known risks, and long-term monitoring with the treatment team.