U.S.
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.
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.
Source: NPR News — Original report ↗
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Original source: NPR News ↗