How one family gave a critical boost to gene therapy research
Summary
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.
Description from NPR.
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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.
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