Uniting families – and their voices – to drive change for those living with Duchenne.
To overcome a disease that is as formidable as Duchenne muscular dystrophy, we need all the help we can get from people who truly care about making life better for our children and young adults. Join us as we press for timely access to affordable treatments in Canada.
From Duchenne Awareness Month in September through to advocacy workshops, our organization prioritizes work that moves the needle forward to enhance Duchenne care and timely access to affordable treatments. Our organized efforts, including lobbyism and policy reform, increase international awareness surrounding the disease and the role of government and private institutions in eradicating its life-limiting impact.
Why do we need advocacy to defeat Duchenne?
- Health Canada’s current framework means drug and therapeutic treatment approval remains inflexible.
- Treatments have been implemented around the world that remain inaccessible to Canadian patients.
- In the case of the Covid-19 vaccine, we have seen the opportunities inherent in “fast-tracking” life-saving therapies and must mobilize this momentum.
- Currently, patient input for rare diseases is not included in the funding and approval process for treatments and therapies. This involvement is critical to developing a pathway to participation, especially within processes that need to be totally transparent.
Advocacy is the only way to transform systems that prevent children with Duchenne from growing into healthy adults.
"There is hope on our horizon with a number of drugs in the pipeline, some of these approved in other countries. But hope is hard to hold on to while we wait for these treatments to be approved in Canada. We need access to therapies approved to be safe and effective now before it’s too late."
- Nicola Worsfold (Mother of Owen, living with Duchenne)
National Vice-President, Research, Education and Advocacy
at Defeat Duchenne Canada
Our advocacy priorities include:
- Work with government agencies to enhance access to therapies.
- Lobby for improved decision-making models to remove barriers to safe and effective medicines for Canadian patients.
- Refute price restrictions imposed by the Patented Medicine Prices Review Board (PMPRB) to ensure medications are affordable and accessible in Canada.
- Nurture pivotal media partnerships to raise awareness regarding rare diseases and the systemic inequities that deny access to families in need.
- Host events that bring together government officials, researchers and families to nurture understanding of our shared goals and ignite fruitful collaborations.
We are proud of our Canadian healthcare system, and we readily see the immense benefits of how it operates. Our organization, its researchers and clinicians recognize the importance of due process and the ways these checks and balances protect patients. However, we also know our American and European counterparts are continuously given access to safe and effective therapies long before there is even a whisper of approval closer to home.
When you are the parent of a child who has lost their ability to walk and will, undoubtedly, also lose their life, the thought you are being denied the ability to do absolutely everything in your power to save them is debilitating. Many of the parents we work with know there are therapies that may slow the progression of Duchenne and enhance their child’s quality of life – yet, they cannot take advantage of these innovations due to convoluted processes.
What we’ve achieved together:
Alongside leaders in rare disease advocacy, Duchenne research and care, we have strategically mobilized this parental passion for transforming policy and bringing hope of progress across borders. Read more about the impact we’ve made through our advocacy work to date:
Become an Advocacy Ambassador:
Help educate our nation’s leaders to get the federal support needed to make real progress in Duchenne trials, treatments and care. Join our national network of Advocacy Ambassadors and learn how you can take action in your community.