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NPR reports that the FDA approved a gene therapy for Sanfilippo syndrome after a family affected by the condition raised millions of dollars for research. The article summary does not describe the therapy’s benefits, risks or approval conditions.
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The approval is relevant to people affected by Sanfilippo syndrome and their families, though the summary does not say how many people may be eligible or when treatment will be available. U.S. approval is a direct development for patients and health care providers, but practical access details are not provided.