voretigene neparvovec (Luxturna) Report issue

Gene therapy Orphan Drug FDA Approved FDA Breakthrough Therapy FDA

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Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).   Wikipedia

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