Bridging the gap in treatments, policies, and support for Duchenne and all rare disease families in Canada.
On February 28th, we recognize Rare Disease Day. For families impacted by Duchenne muscular dystrophy and other rare diseases, it is a day to raise awareness of what is happening in Canada for people with rare diseases.
Thirty years ago, John Davidson set out on a journey that would forever change the landscape of Duchenne in Canada and across the world. His son, Jesse, was diagnosed with this devastating rare disease defined by weakness in skeletal, lung, and heart muscles. John did what any parent would do – he fought. He walked across Ontario, nearly 3,500 kilometres, step by powerful step, refusing to let Duchenne take his son’s future without a fight.
Closing the Treatment Gap
Eight Duchenne-specific therapies are available in the United States and other regions, yet not a single treatment available in Canada. This is not surprising; only 67% of any new medication approved in the U.S. or Europe reaches Health Canada for review, and the gap is worse for rare disease treatments (Gaudette 2023).
That is why Rare Disease Day is so critical. It is a day to bring attention to the issues that face so many Canadians today. The Government of Canada’s National Strategy for Drugs for Rare Diseases was seen as a positive first step. Now, nearly two years later, as these funds are being dispersed to provinces, it will be vital to ensure that dollars are spent to provide sustainable benefits for as many families affected by rare diseases as possible.
A rare disease diagnosis isolates families, forcing them to navigate a system that often feels indifferent to their urgency. This Rare Disease Day, we mark another year of hope, resilience, and the determination of rare disease families who wake up every day with the same quiet questions: Will my child be safe tonight? How much more time do we have? How many more summers?
This Rare Disease Day, we mark another year of hope, resilience, and the determination of rare disease families who wake up every day with the same quiet questions: Will my child be safe tonight? How much more time do we have?
How many more summers?
Not Just Another Promise
For three decades, Defeat Duchenne Canada has fought with Duchenne families for care, treatments, and policies. The fight against Duchenne is part of a larger battle affecting all rare disease families. We must ensure that government leaders hear our voices, understand our urgency, and invest in meaningful solutions.
This strategy must become a lifeline for those impacted by rare disease. It must build a future where parents no longer have to wake up each day
wondering if their child will have access to the care they deserve.
We call on Health Canada to ensure that this investment into rare diseases leads to true, tangible, lasting outcomes for rare disease families. Let’s change the future so that in 2025, Canadians with Duchenne and other rare diseases with treatments available in other regions will no longer have to wake up each day wondering if they will have access to the care they deserve.
Take Action Against Rare Diseases
This Rare Disease Day, consider writing to your government official to tell them how you are impacted by a rare disease, and how you want to see them take a stand. The time is now, and for those impacted by Duchenne, time is muscle!
Learn more about Duchenne and how you can get involved this Rare Disease Day
defeatduchenne.ca/news-and-events/events/rare-disease-day-2025/.
