How one family gave a critical boost to gene therapy research | HappeningNow.news

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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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A family affected by Sanfilippo syndrome raised millions of dollars for research. The Food and Drug Administration recently approved a gene therapy that the family’s support helped make possible.

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