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

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). Pre...

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Bibliographic Details
Main Authors: Fred K Chen, Alex W Hewitt, Alan Ma, John Grigg, Keith R Martin, Robyn Jamieson, Heather G Mack, Fleur O’Hare, David Mackey, John De Roach, Alexis Ceecee Britten-Jones, Myra McGuinness, Nicole Tindill, Lauren Ayton, Anai Gonzalez Cordero, Thomas L Edwards, Gladys Ho, Michael Hogden, Anthony Kwan, Tina Lamey, Terri McLaren, Benjamin Nash, Jon Ruddle, Matthew Simunovic, Ingrid Sinnerbrink, Deepa Ajay Taranath, Jen Thompson, Jaclyn White
Format: Article
Language:English
Published: BMJ Publishing Group 2021-06-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/11/6/e048361.full

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https://bmjopen.bmj.com/content/11/6/e048361.full

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