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.
An FDA advisory panel gave the green light for a therapy that can improve the vision of people with a rare genetic disorder called Leber's congenital amaurosis (LCA)
The treatment targets a rare form of childhood blindness caused by a defective gene.
If approved, it would be the first gene therapy in the U.S. for an inherited disease
CNBC's Meg Tirrell reports on Spark Therapeutics shares jumping after the FDA panel votes in favor of the company's gene therapy.
The pioneering treatment fixes a mutated gene and could soon be available in the U.S.
SILVER SPRING, Md. (AP) — The Latest on an FDA panel's discussion on a gene therapy treatment for blindness (all times local):; 3:19 p.m. U.S. health advisers have endorsed an experimental approach to treating inherited blindness, setting…
SILVER SPRING, Md. (AP) — A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval Thursday, as federal health advisers endorsed the experimental gene therapy for patients with an inherited…