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How one family gave a critical boost to gene therapy research

NPR News •
November 01, 2026 • 10:00 AM
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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.

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Same Story from Other Publications

Compare how 1 other independent news outlet reported on this story.

1 Publication
Sydney Morning Herald (SMH)
2h ago
Out of the bubble: How a world-first gene therapy gave Matthew a second chance at life

Out of the bubble: How a world-first gene therapy gave Matthew a second chance at life

Until recently, Matthew Rozek has spent his life avoiding everyday germs. This has meant avoiding parties, shopping malls, flights, and public transport. But a new gene therapy treatment has given him a new lease on life.

#gene #therapy
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