Jesse’s Journey, Stand for Duchenne Canada and La Force DMD have come together with the Duchenne muscular dystrophy (DMD) community to voice our concern regarding the current lack of Health Canada-approved treatment options for Canadians living with this debilitating and life-limiting rare disease. Despite the fact that new therapies – Translarna (ataluren) and Exondys 51 (eteplirsen) – have been approved in Europe and the United States for several years now, in Canada we are still waiting. There is an urgent need to implement an accelerated pathway for rare disease drug approvals that includes input in the form of real-world input and lived patient and caregiver experience. We acknowledge the work Health Canada is currently undertaking to modernize its approaches regarding drug approval, access to treatments and international collaboration – but the timelines to make these changes are too long. The harsh reality is that any child diagnosed with Duchenne around the time that treatments such as Translarna (ataluren) were initially ready to be evaluated by Health Canada, is most likely no longer able to walk – and may be starting to experience life-threatening respiratory or cardiovascular issues. This is why we need to work together to defeat Duchenne now – there is no time to wait. We as a Duchenne community need our Canadian regulatory bodies to know why we can’t wait any longer for these treatments and understand the impact the lack of approved treatments is having on our Canadian children and young adults. Please help us to ensure that federal elected representatives – Members of Parliament (MPs) – and decision makers understand that all treatments for Duchenne considered to be safe and effective, need to be made available in this country as quickly as possible.