Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy

Author:

Kang Connie,Scott Lesley J.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology,Genetics,Molecular Medicine,General Medicine

Reference31 articles.

1. Motta FL, Martin RP, Filippelli-Silva R, et al. Relative frequency of inherited retinal dystrophies in Brazil. Sci Rep. 2018;8(15939):1–9.

2. Ziccardi L, Cordeddu V, Gaddini L, et al. Gene therapy in retinal dystrophies. Int J Mol Sci. 2019;20(5722):1–25.

3. National Institute for Health and Care Excellence (NICE). Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations [ID1054]: pre-briefing meeting. 2018. https://www.nice.org.uk/guidance. Accessed 29 May 2020.

4. European Medicines Agency. Luxturna: EU assessment report. 2019. https://www.ema.europa.eu. Accessed 29 May 2020.

5. Spark Therapeutics. LUXTURNA (voretigene neparvovec-rzyl) intraocular suspension for subretinal injection: US prescribing information. 2019. https://www.fda.gov. Accessed 29 May 2020.

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