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A family's fundraising for gene therapy research pays off

NPR | By Sydney Lupkin
Published October 22, 2026 at 4:55 PM EDT

After their daughter was diagnosed with a rare inherited disease in 2013, the O'Neill family raised money to find a treatment. The Food and Drug Administration recently approved a gene therapy.

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Sydney Lupkin
Sydney Lupkin is the pharmaceuticals correspondent for NPR, where she has reported on accelerated drug approvals whose confirmatory trials were overdue, Operation Warp Speed contract accountability, and the ripple effects of obesity drug shortages. 
See stories by Sydney Lupkin