Dear Canadian Duchenne Community,

Today, we share difficult news that weighs heavily on all of us. PTC Therapeutics has announced that they will be closing the ataluren clinical trial. This means that families in Canada who have been accessing ataluren through the clinical trial will no longer be able to receive this medication.

We know how devastating this feels and this is not the first time this has happened to our Canadian families. For many, ataluren represented hope at a time when treatment options in Canada remain extremely limited. Some of you have invested years travelling long distances for study visits, rearranging your lives around trial participation, or navigating the complexities of accessing this therapy. You did all of this out of love, commitment, and belief in giving your child every possible chance. We see you, and we honour the sacrifices you have made.

While this decision aligns with regulatory actions in other countries, it does not lessen the impact on families here at home. We also recognize that this was not an easy decision for PTC. They have dedicated significant time, scientific effort, and financial resources over many years to develop and evaluate this therapy. Unfortunately, the harsh reality in rare disease research is that clinical research programs cannot continue indefinitely. Which is why we Canadians living with Duchenne urge our government to prioritize access to promising new treatment approaches to treat Duchenne muscular dystrophy in Canada. 

To the families who have been relying on ataluren, those who believed they were seeing benefit, those who felt they were finally able to do something, we know this loss is profound. Losing an option, when there is nothing else to fall back on is incredibly hard. And this is true for all Canadian families who have been impacted by the closure of a clinical trial.

Please know that Defeat Duchenne Canada remains steadfast in our advocacy. We will continue fighting for timely and equitable access to treatments for all Canadians living with Duchenne. Time is muscle and urgency matters. Now more than ever, we need to stand together as a unified community to ensure our governments understand the importance of choice, the value of emerging therapies, and the critical need for a clear, fair pathway for access to medicines for Duchenne muscular dystrophy.

Our commitment to you has not wavered. We will continue to push for better options, better systems, and better futures for all families affected by Duchenne.

With sincerity,

Nicola Worsfold
Executive Director and Duchenne Mom
Defeat Duchenne Canada