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24. Retinal Dystrophies and the Road to Treatment: Clinical Requirements and Considerations. Talib M; Boon CJF Asia Pac J Ophthalmol (Phila); 2020; 9(3):159-179. PubMed ID: 32511120 [TBL] [Abstract][Full Text] [Related]
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26. Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl. Bennett J; Maguire AM Cold Spring Harb Perspect Med; 2023 May; 13(5):. PubMed ID: 36167727 [TBL] [Abstract][Full Text] [Related]
27. Voretigene neparvovec-rzyl (Luxturna) for inherited retinal dystrophy. Med Lett Drugs Ther; 2018 Mar; 60(1543):53-55. PubMed ID: 29635265 [No Abstract] [Full Text] [Related]
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30. The pros and cons of vertebrate animal models for functional and therapeutic research on inherited retinal dystrophies. Slijkerman RW; Song F; Astuti GD; Huynen MA; van Wijk E; Stieger K; Collin RW Prog Retin Eye Res; 2015 Sep; 48():137-59. PubMed ID: 25936606 [TBL] [Abstract][Full Text] [Related]
31. The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene. Chung DC; Bertelsen M; Lorenz B; Pennesi ME; Leroy BP; Hamel CP; Pierce E; Sallum J; Larsen M; Stieger K; Preising M; Weleber R; Yang P; Place E; Liu E; Schaefer G; DiStefano-Pappas J; Elci OU; McCague S; Wellman JA; High KA; Reape KZ Am J Ophthalmol; 2019 Mar; 199():58-70. PubMed ID: 30268864 [TBL] [Abstract][Full Text] [Related]
32. Full-field Scotopic Threshold Improvement after Voretigene Neparvovec-rzyl Treatment Correlates with Chorioretinal Atrophy. Stingl K; Stingl K; Schwartz H; Reid MW; Kempf M; Dimopoulos S; Kortuem F; Borchert MS; Lee TC; Nagiel A Ophthalmology; 2023 Jul; 130(7):764-770. PubMed ID: 36822437 [TBL] [Abstract][Full Text] [Related]
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