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)
Author: Source Author
Source: NPR.
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.
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