Time is Muscle for Children Living with Duchenne Muscular Dystrophy

Rochelle Ten Haaf

National Director, Marketing & Stakeholder Engagement, Defeat Duchenne Canada

Six medications have been approved for Duchenne muscular dystrophy in the United States and Europe, while Canadians have nothing. 

One in every 5,000 boys worldwide is born with Duchenne, the most common fatal form of muscular dystrophy. The disease is relentless. It slowly weakens the body’s muscles, deteriorating the function of vital organs and ultimately – shortening their life. 

Currently, six medications are approved in the United States and Europe to slow the progression of this muscle-wasting disease, yet nothing has been approved in Canada.  

In 2019, the federal government committed $1 billion to set up a national Rare Disease Drug Strategy, with an additional $500 million annually. Three years later, after numerous consultations led by the Canadian Organization for Rare Disorders (CORD), we are still waiting for the federal government to implement a Rare Disease Strategy that supports a Rare Disease Drug Framework. Time is muscle, and the lives of our Canadian children and young adults with Duchenne are at risk. 

“We are proud of our Canadian healthcare system, and we readily see the immense benefits of how it operates. Our organization, researchers and clinicians recognize the importance of due process and how these checks and balances protect patients. However, we also know our American counterparts are continuously given access to safe and effective therapies long before there is even a whisper of approval closer to home,” shares Perry Esler, CEO of Defeat Duchenne Canada – the country’s only national charity dedicated to ending Duchenne muscular dystrophy. 

“When you are the parent of a child who has lost their ability to walk and will, undoubtedly, also lose their life, the thought that 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. Alongside leaders in Duchenne research, we strategically mobilize this parental passion to transform policy and bring the hope of progress across the border.”  

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, National Vice-President, Research, Education and Advocacy at Defeat Duchenne Canada (Mother of Owen, living with Duchenne)

Please join the fight to help ensure all treatments for Duchenne muscular dystrophy deemed safe and effective are made available in our country as quickly as possible.  

Click here to write your local member of parliament on February 28, Rare Disease Day, highlighting the need for timely access to Duchenne treatments in Canada.