Yesterday, Roche/Sarepta announced that the FDA has lifted its pause on shipments of delandistrogene moxeparvovec for ambulatory individuals living with Duchenne. This is very encouraging news for the global Duchenne community, and we join in relief and celebration alongside those outside of Canada who may now move forward with access. At the same time, this update draws attention to the persistent uncertainty Canadians face in accessing emerging therapies—an issue that weighs heavily on our families and loved ones.
We recognize how sensitive and emotionally complex this news is for our Canadian Duchenne community. While others begin accessing potentially life-changing treatments, we remain in a position of waiting with few options available, and the weight of watching from the sidelines. For Canadians, clinical trials remain the sole path to access and are an option weighed with significant emotional and mental burden, complex decisions, and unknown risks. It is an incredibly difficult position to be in.
We encourage you to speak openly with your clinical care teams about any questions or concerns you may have regarding emerging therapies or trial participation.
Defeat Duchenne Canada remains committed to advocating for equitable and timely access to new therapies in Canada. If you’d like to learn more about our efforts or join us in advocating for change, please reach out.
You can view Roche’s letter to the community by clicking on the link below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: