Journal article

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

Heather G Mack, Fred K Chen, John Grigg, Robyn Jamieson, John De Roach, Fleur O'Hare, Alexis Ceecee Britten-Jones, Myra McGuinness, Nicole Tindill, Lauren Ayton

BMJ OPEN | BMJ PUBLISHING GROUP | Published : 2021

Abstract

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). Previous studies have shown that individuals who might participate in gene therapy trials overestimate clinical effect and underestimate risks. However, little is known about the perspectives of patients who may be offered approved gene therapy treatment for ocular conditions (as distinct from participating in clinical trials of gene therapy). The main objective of this study is to develop a tool to..

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Grants

Awarded by Australian National Health and Medical Research Council Centre of Research Excellence grant


Funding Acknowledgements

This work was supported by a Retina Australia medical research grant (to HM, FC, JG and LA) for 2021, grant not numbered. Researchers were also supported by an Australian National Health and Medical Research Council Centre of Research Excellence grant (GNT1116360: DM, FKC), Fellowships (MRF1142962: FKC and MRF1151055: LNA), and Ideas Grant (FKC GNT1188694). Centre for Eye Research Australia receives support from the Victorian Government through its Operational Infrastructure Support ProgramProgramme.