We Don’t Want To Disappear.
Support children and adults with rare diseases now by signing our petition.
We’re not so rare.
Please sign our petition. Share with friends.
Tell our government: it’s time.
In Canada, 1 in 12 people are afflicted with a rare disease. That amounts to 3 million people, and two-thirds are children. Together, people with rare diseases are not so rare. Canada is one of the few developed countries without a national rare diseases strategy to guide diagnosis, treatment and management of rare diseases.
People with rare diseases in Canada, mostly children, miss out on timely and affordable access to new therapies and supportive care that helps them to survive and thrive.
Our national appeal, “Not so rare”, calls on governments to implement a national strategy for rare diseases as fast as possible – because diseases don’t wait!
A national strategy for rare diseases would support a plan for:
- Early diagnosis
- Faster access to promising new therapies
- Fair and equal access to supportive care and effective disease management strategies
- Ending the inefficient, two-tiered, federal-provincial system for drug marketing approval and reimbursement of costs for orphan drugs
A national strategy is the first step to providing the promising new treatments and supportive care that people with rare diseases desperately need.
In November 2019, little Elena and her family participated in our studio recording for the Not So Rare campaign. Elena and her parents were so committed to our cause that they didn’t hesitate to make the return trip between Quebec City and Montreal, more than 4 hours away, to participate in our video shoot. Almost a year later, on November 21, 2020, at 8:15 a.m., Elena took her last breath. We write this article to pay tribute to this courageous family and to raise awareness of the reality of ultra-rare diseases. Alone in the world with her illness Some diseases may affect only a few dozen people in the world. They are said to be “ultra rare”. This is precisely the case with Elena’s disease, a genetic neurodegenerative disease linked to the AGTPBP1 gene. The disease, which does not yet have a name, was discovered in Germany in June 2019, just a few months before Elena’s diagnosis. The little girl was the only registered patient in the world. When a disease is so rare, there are extremely few dedicated resources, and other