How one family gave a critical boost to gene therapy research

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.(Image credit: Robert Clark)

aath
November 1, 2026
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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.

(Image credit: Robert Clark)

Source: NPR Topics: News