Today is truly a meaningful day for our community as we gather united by one shared goal of improving the lives of Canadians affected by Duchenne muscular dystrophy.
Rare Disease Day is more than a date on the calendar. It’s a global call to action. Around the world, communities come together to shine a light on rare diseases and to advocate for equity in healthcare, research, and access to life-changing treatments.
In Canada, 1 in 12 people (that’s approximately 3 million Canadians) live with a rare disease. Duchenne is one of those diseases, and while we’ve seen remarkable scientific progress in recent years, access here in Canada has been at a standstill for far too long. Historically, only about 60% of rare disease treatments ever make it to Canada. And until October of last year, none had reached our Canadian Duchenne community.
That’s why today matters so much to us.
Defeat Duchenne Canada exists to end Duchenne. We are building a community of families, we invest in innovative research with the goal of improving the health and well being of our loved ones, we provide education and support to our families and individuals affected by Duchenne, and advocate to ensure our community has equitable, timely, and affordable access to the best treatments and care.
This Rare Disease Day, we are proud to stand alongside the global movement focused on taking action.
And very timely too, this past week we just received a positive draft recommendation from the Canadian Drug Agency for vamorolone (now known as AGAMREE). This is a big win and a significant milestone for Canadians living with Duchene, and a testament to the strength and persistence of our community. We are currently preparing feedback on the reimbursement criteria, and we’re hopeful that our input will help shape the final recommendation.
But this is only the beginning.
The next critical step in accessing this medication though our public health care system now falls to the provincial level. Each province must now decide whether to reimburse AGAMREE, and this is where your voice becomes essential.
Our goal for today is to empower you with the tools, knowledge, and confidence to take action, and we will do that by walking you through our advocacy toolkit developed specifically for our Duchenne community, and how you can engage decision-makers effectively.
Rare Disease Day reminds us that while each disease may be rare, together we are a powerful community, and when we raise our voices together, change happens.
Thank you for being here, and for being part of this movement.
Nicola Worsfold
Executive Director & Duchenne Mom
Defeat Duchenne Canada