Nicola Worsfold
Executive Director of Defeat Duchenne Canada
Canada’s Duchenne community is advancing research, but regulatory and access gaps continue to delay promising therapies for families.
For the approximately 800 to1,000 Canadians living with Duchenne muscular dystrophy, a rare, progressive neuromuscular disease, the gap between scientific progress, regulatory approval and access can have profound consequences. Since 1995, Defeat Duchenne Canada has invested $20.3 million in research worldwide to advance treatments, improve care and ultimately find a cure.
Canada is a long-standing leader in Duchenne research, with Canadian researchers, patients, and families playing an important role in international clinical trials that have contributed to the development of multiple new therapies approved in other countries. Yet, as of October 2025, only one therapy has been approved by Health Canada. This inequity highlights the disconnect between Canada’s research leadership and timely, equitable access to the treatments Canadian families have helped to advance.
Building a pathway to better access
“As a Duchenne mom, I know first hand that families participate in research with hope; not only for their own children, but for every family living with Duchenne,” says Nicola Worsfold, Executive Director of Defeat Duchenne Canada. “We are committed to working with researchers, governments and industry to ensure that Canadians can benefit from the therapies they have invested their blood, tissue and time to make possible.”
The National Strategy for Drugs for Rare Diseases and Health Canada’s regulatory modernization efforts create an opportunity to reduce delays and improve access to treatment. Now is the time for Canada to build on this momentum to ensure promising Duchenne therapies reach Canadian families.
To learn more visit www.defeatduchenne.ca.
