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)
How one family gave a critical boost to gene therapy research
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NPR NewsBy Sydney Lupkin
EXECUTIVE 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.
Key Takeaways
- 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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Published originally on NPR News
