A first-of-its-kind gene therapy received approval from the Food and Drug Administration on Tuesday to treat a rare, inherited form of childhood blindness.
The FDA marketing clearance of Spark Therapeutics’s Luxturna is historic for scientific and financial reasons. Luxturna is the first gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.
This is just for RPE65 correct? I have 2 boys with retinitis pigmentosa. My fiancées boy was one of the youngest to undergo the gene therapy for LCA. Corey Haas.
A good start
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