FDA Approves Gene Therapy Backed by Sanfilippo Family’s Fundraising
A family affected by Sanfilippo syndrome raised millions for research that helped make a newly FDA-approved gene therapy possible.
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The Food and Drug Administration has approved a gene therapy that a family affected by Sanfilippo syndrome helped make possible through fundraising, NPR reported.
The family raised millions of dollars to support research. That funding provided a critical boost to work behind the newly approved treatment.
Originally reported by NPR. Read the full story at NPR
