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FDA advisers review gene therapy to treat form of blindness

In this Oct. 4, 2017 photo, Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. Misa was 4 years old when he received his gene therapy treatment. (AP Photo/Bill West)

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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