Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey

INTRODUCTION: Voretigene neparvovec-rzyl (Luxturna) was approved by the Australian Therapeutic Goods Administration on 4 August 2020 for the treatment of biallelic mutations in the RPE65 gene, a rare cause of congenital and adult-onset retinal dystrophy (predominantly Leber congenital amaurosis). Pr...

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Detalles Bibliográficos
Autores principales: Mack, Heather G, Chen, Fred K, Grigg, John, Jamieson, Robyn, De Roach, John, O’Hare, Fleur, Britten-Jones, Alexis Ceecee, McGuinness, Myra, Tindill, Nicole, Ayton, Lauren
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2021
Materias:
Ophthalmology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8220456/
https://www.ncbi.nlm.nih.gov/pubmed/34158306
http://dx.doi.org/10.1136/bmjopen-2020-048361

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