Case study: Gene therapy for inherited retinal disease

At the December 2021 conference held by the Canadian Organization of Rare Disorders (CORD), FBC's president & CEO Doug Earle was joined by Dr. Elise Héon and patient advocates Jack and Sitka to discuss gene therapy for inherited retinal disease.

In October 2020, Health Canada approved Luxturna as the first therapy for previously untreatable inherited retinal disease. This one-time therapy replaces dysfunctional RPE65 genes with working copies that restore vision and improve sight.

In Canada, for children and adolescents with IRD, there is an urgent need for timely access to Luxturna, with several young people moving beyond the period of efficacy now at high risk of losing their vision. Despite a CADTH positive recommendation for funding, the provinces have not yet provided access. Private payers have denied access, citing pre-existing condition.
This situation must be addressed with a national solution in the future Rare Disease Drug Strategy. In the meantime, we urgently need intervention to save the sight of children who are NOW at risk.

To add your voice to accelerate access to Luxturna for Canadians, send an email by visiting https://approveluxturna.ca/act-now/

To learn more about Fighting Blindness Canada please visit https://www.fightingblindness.ca/

To learn more about the Canadian Organization of Rare Disorders (CORD) please visit https://www.raredisorders.ca/

STAY CONNECTED
Twitter: https://twitter.com/FB_Canada​
Facebook: https://www.facebook.com/FightingBlin​...
Instagram: https://www.instagram.com/fightingbli​...
Sign-up for our monthly e-newsletter: https://www.fightingblindness.ca/enew​...

CONTACT US
General Inquiries: [email protected]
Health Information Line: 1.888.626.2995 | [email protected]

ABOUT FIGHTING BLINDNESS CANADA Fighting Blindness Canada (FBC) is Canada’s leading private funder of vision research. We offer hope to Canadians by identifying the best, most promising research that is driving treatments and cures for blinding eye diseases, and by raising and stewarding funds to support this essential, sight-saving research. All of the research and educational initiatives we fund supports our goals of understanding why vision loss occurs, how it can be slowed or stopped, and how sight can be restored. Learn more about Fighting Blindness Canada: https://www.fightingblindness.ca/abou​...