NPR

FDA Panel Endorses Gene Therapy For A Form Of Childhood Blindness

Food and Drug Administration advisers unanimously recommended that the agency approve the first gene therapy for an inherited disease — a rare defect that causes blindness in children.
A panel of experts has recommended that the Food and Drug Administration approve a treatment developed by Spark Therapeutics for a rare form of blindness. Source: Spark Therapeutics

Gene therapy, which has had a roller-coaster history of high hopes and devastating disappointments, took an important step forward Thursday.

A Food and Drug Administration advisory committee endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood.

The recommendation came in a unanimous 16-0 vote after a daylong hearing that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by the treatment.

"Before surgery, my vision was dark. It was

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