Defeat Duchenne Canada is proud to share a historic milestone for the Canadian Duchenne community: Health Canada has approved AGAMREE® (vamorolone) as the first treatment for Duchenne muscular dystrophy (DMD) in Canada.

This long-awaited approval represents the result of years of research and collaboration, with families, researchers, clinicians, and Defeat Duchenne Canada working together to advance treatment options available to Canadian Duchenne families. AGAMREE®  is an oral therapy designed as an alternative to the current standard of care with fewer side effects, including reduced impact on growth, bone health, and behaviour. AGAMREE®is approved for the treatment of Duchenne Muscular Dystrophy in boys aged 4 and up. See more details in the full press release from Kye Pharmaceuticals Inc. below. 

Defeat Duchenne Canada is paving the road to progress: In 2020, Defeat Duchenne Canada awarded a research grant to fund a Canadian clinical trial that included a broader age range for boys ages 2–4 and 7–17, with multiple sites across Canada. The commitment of Canadian families who participated in these trials provided the critical data that supported Health Canada’s decision.

John Davidson, Founder of Jesse’s Journey and current board member of Defeat Duchenne Canada, reflected: “It’s very impactful knowing that the first medicine approved in Canada is one that Defeat Duchenne Canada supported through research.” 

Jesse’s legacy continues through this milestone, a testament to the power of community and perseverance. Learn more about Jesse and the history of Defeat Duchenne Canada at defeatduchenne.ca/our-history.

Your dollars make a difference, and we want to thank the generosity of our donors. Your support helps us fund the research that can make a significant impact on the lives of individuals living with Duchenne in Canada.

What This Means for Families

“For families like ours, this news means more than words can say. It gives us hope where there has so often been fear and uncertainty. Knowing there is finally a treatment approved in Canada definitely brings some sense of relief,” shared Anna Vockeroth, a Canadian Duchenne mom.

The Road Ahead: Equitable Access

While today is a day of celebration, Defeat Duchenne Canada emphasizes that the work is far from finished. Approval is only the first step. 

“This is an exciting day for Canadians and I join many Duchenne parents in celebrating our very first treatment approved in Canada. It is thanks to the collaborative efforts of all of those who have been involved in bringing this treatment to fruition. My hope is that this is the first of many treatment options that will become available to Canadians living with Duchenne,” said Nicola Worsfold, Executive Director of Defeat Duchenne Canada.

The organization will now shift focus on ensuring equitable, affordable, and timely access for families who could benefit from AGAMREE® across every province and territory.

Lend your voice to Defeat Duchenne Canada’s advocacy efforts. Together, our collective voices are the driving force for change.

Moving Forward Together

This milestone is a powerful reminder of what we can achieve when we work together. 

“This has been a year of celebration for Defeat Duchenne Canada. We are thrilled to share multiple milestones achieved in 2025: our 30th anniversary, John’s return to the road, and now we celebrate the first approved treatment for Duchenne in Canada,” said Nicola Worsfold, Executive Director of Defeat Duchenne Canada.

We extend heartfelt gratitude to the families, donors, clinicians, researchers, and partners who have stood with us on this journey. This progress belongs to all of us. Together, we will continue to raise your voices until every family across Canada has access to life-changing treatments and until we achieve our shared vision: a future free from Duchenne.

Register for the Family Forum to connect directly with the researchers leading the fight against Duchenne at defeatduchenne.ca/familyforum.