Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease
Open Access
- 1 October 2019
- journal article
- research article
- Published by American Medical Association (AMA) in JAMA Ophthalmology
- Vol. 137 (10), 1115-1123
- https://doi.org/10.1001/jamaophthalmol.2019.2512
Abstract
Biallelic RPE65 (OMIM *180069) mutation-associated inherited retinal disease (IRD) is an ultrarare, severely progressive retinal condition that affects approximately 1000 to 2000 patients in the United States.1-8 Because this disease has been considered medically untreatable, management has focused on psychological support and visual rehabilitation, including low vision aids.Keywords
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