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FDA advisers review gene therapy to treat form of blindness

U.S. health advisers are deciding whether to recommend approval of an experimental treatment for improving vision in patients with a rare form of blindness.

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Thursday, October 12, 2017

SILVER SPRING, Md. (AP) — U.S. health advisers are reviewing a novel treatment for a rare form of inherited blindness, potentially setting the stage for the launch of a groundbreaking new genetic medicine.

The Food and Drug Administration panel will vote Thursday on whether to recommend approval of Philadelphia-based Spark Therapeutics’ treatment, which aims to improve vision in some patients with hereditary blindness by replacing a defective gene that affects vision.

The FDA is not required to follow the group’s recommendation, but such deliberations are generally the final step in the agency’s decision-making process. The FDA has until mid-January to decide. If approved, it would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of related conditions.

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