You have been directed to this as a client of Vision Australia on behalf of the Centre for Eye Research Australia (CERA) at the University of Melbourne.
Voretigene (Luxturna™) has been approved by the Australian Therapeutic Goods Administration for use as gene therapy in adults and children with visual loss due to one of the causes of retinitis pigmentosa - mutations in the RPE65 gene. A start date is projected for later in 2021.
Retinal degeneration caused by mutations in RPE65 is very rare. These gene mutations are responsible for about 10 per cent of cases that arise in infancy (known as Leber congenital amourosis) and about two per cent of cases that arise in adults (retinitis pigmentosa). This means that the majority of patients with retinal degeneration/retinitis pigmentosa will not have mutations in RPE 65 gene and will not benefit from this new treatment. It is anticipated the voretigene treatment would be appropriate for around one to two patients each year in Australia.
You are invited to complete a survey on your knowledge and expectations regarding future gene therapy trials for inherited retinal dystrophy, as well as voretigene treatment for patients with RPE65 gene mutations.
You will read an on-line consent form and then continue to the survey proper. At the end of the survey you can indicate if you would like to be contacted for possible testing to see if you have the mutation in RPE65 or any other identified mutations associated with inherited retinal dystrophy, receive a lay-person summary of the results of the study when it is finished and if you consent to having your de-identified responses stored for possible future research (subject to further ethics approval).
Please note that your personal details have remained confidential with Vision Australia and all survey participants’ personal details will be confidential. All data will be anonymised and you will not be identifiable within any publications made as a result of this study.
Thank you for participating in the survey. Please click here to proceed to the survey (or type https://www.cera.org.au/ird-survey/ into your browser).
Yours sincerely,
Heather G Mack (principal researcher), Lauren Ayton (responsible researcher), Fred C Chen, John Grigg, Thomas Edwards, Fleur O’Hare, Ceecee Zhang, Keith Martin – University of Melbourne.
For further assistance or information about the survey from CERA, call (03) 8344 3441. For more information about Vision Australia and our specialist blindness and low vision services, call 1300 84 74 66 or email [email protected].