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How one family gave a critical boost to gene therapy research

NPR News, Sun Nov 1, 2:00 AM MST (0900Z)

A family affected by Sanfilippo syndrome raised millions of dollars for research. The Food and Drug Administration just approved a gene therapy the support helped make possible.

https://www.npr.org/2026/11/01/nx-s1-6014739/sanfilippo-gene-therapy-fayuvi