Will the newly FDA approved gene therapy help my child?
On December 19, 2017, the FDA approved for the first time a gene therapy product for a type of congenital retinal blindness, Leber Congenital Amaurosis caused by mutations in both copies of the RPE65 gene. Most patients with RPE65-related inherited retinal disease have poor vision from birth, but some patients lose vision at older ages. Many patients and parents wonder if they or their child might benefit from this new treatment. The approved gene therapy product, Luxturna®, is an adeno-associated viral vector carrying a normal copy of the human RPE65 gene. It is injected under the retina into the subretinal space following a vitrectomy (surgical removal of the vitreous). This procedure is performed under general anesthesia. This treatment will be available at several sites across the country. Because this is a brand new therapy, insurance coverage issues currently are not known. Some frequently asked questions and answers are provided below. Parents and patients can also be referred to the AAPOS Genetic Eye Disease Task Force members who have posted contact information on the AAPOS website.
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