We write regarding the administration of the Food and Drug Administration’s (FDA’s) Pediatric Rare Disease Priority Review Voucher (PRV) program, particularly the decision by the previous administration to deny a PRV for a one-time administered novel curative gene therapy that treats sickle cell disease.
Tim Scott: “We write regarding the administration of the Food and Drug Administration’s (FDA’s) Pediatric Rare Disease Priority…”
Editor's note · Context
Sen. Scott urges FDA to reconsider gene therapy decision.
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