Turning Awareness into Action for Duchenne
Rare Disease Day, observed internationally on February 28, is more than a moment of awareness – it is a call to action. Each year, it brings together families, advocates, healthcare professionals, and decision-makers to advance equity in care, research, and treatment access for people living with rare diseases.
In Canada, approximately 1 in 12 people live with a rare disease, including Duchenne muscular dystrophy. Duchenne is a severe, progressive, and life-shortening neuromuscular disorder. For families living with Duchenne, time matters. Irreversible muscle weakness worsens with each passing day, making delays in access to treatment especially devastating.
This Rare Disease Day, Defeat Duchenne Canada is calling on communities across the country to come together and fight for affordable access to treatments in Canada.
Join our Rare Disease Day webinar on Saturday, February 28 from 12:00-1:00 p.m. ET.
A Milestone Moment — With Work Still Ahead
In October 2025, Health Canada approved AGAMREE (vamorolone) the first-ever treatment for Duchenne muscular dystrophy, marking a historic milestone for families who have waited decades for progress. For many, this approval represented hope and validation that scientific advances are finally beginning to reach those who need them most.
However, approval is only the first step in a lengthy process that can take years.
Reimbursement decisions are still required to ensure that every Canadian who wants and needs this treatment can access it. Without timely funding, families remain unable to benefit from a therapy that is available, yet out-of-reach.
With more than eight Duchenne treatments approved in other countries, including the United States and parts of Europe, Canadian families continue to wait. These delays are not abstract, they are measured in declining mobility, lost independence, and reduced quality of life.
Why Advocacy Is Essential
Families living with Duchenne know this fight well. Alongside the emotional and physical toll of the disease, families face financial strain from medical equipment, home modifications, and services not covered by public healthcare.
That is why advocacy matters.
Approval is just the beginning. Families living with Duchenne muscular dystrophy need access to medicines now. With more than eight treatments available internationally, Canadian’s living with Duchenne cannot be left behind.
– Nicola Worsfold, Executive Director at Defeat Duchenne Canada
Rare Disease Day provides a powerful opportunity for families and allies to raise their voices and push for timely, equitable, and affordable access to treatments and care. When communities come together, policy change becomes possible.
Join the EMPOWER Advocacy Webinar
To mark Rare Disease Day, we invite you to participate in the third and final session of our EMPOWER Advocacy webinar series:
📅 Saturday, 28 February, 2026
📍 Virtual
🕛 12:00–1:00 p.m. EST
This practical, action-focused session will equip you to:
- Activate your advocacy skills with confidence
- Use your advocacy toolkit effectively
- Prepare for and conduct meaningful meetings with decision-makers
- Navigate challenges and sustain advocacy momentum
Rare Disease Day reminds us that progress happens when awareness leads to action. By joining this webinar, you are adding your voice to a global movement for fairness, equity, and access to treatments and care, and helping drive change for Canadians living with Duchenne.
Register now to join Defeat Duchenne Canada’s Rare Disease Day advocacy webinar and help drive change for equitable access to Duchenne treatments.
