Defeat Duchenne Canada Provides Feedback on Canada’s Drug Agency Caregiver Guidelines

On April 7, Defeat Duchenne Canada submitted feedback on the Canada Drug Agency’s (CDA) draft Guidance for Incorporating Impacts on Informal Caregivers and Productivity Outcomes in Economic Evaluations, as part of a public consultation. The objective of the guidance is to help make sure decisions about new treatments consider not just patients, but also the impact on caregivers and daily life, including work. By looking at the bigger picture, it aims to support fairer and more informed decisions about access to new therapies. CDA will publish its final guidance later in 2026. 

Update on Canada’s Drug Agency Review of vamorolone (AGAMREE)

Defeat Duchenne Canada also provided feedback on CDA’s draft recommendation for vamorolone to help ensure equitable access for people with Duchenne. Although the draft recommendation was generally positive, the manufacturer requested a reconsideration, which CDA accepted. The expert committee will review this on September 23. While the process has taken longer than expected, we welcome the reconsideration and hope it will broaden access to this first approved Duchenne therapy in Canada. Updates will be shared as more information becomes available.

INESSS Final Recommendation for vamorolone (AGAMREE)

On April 29, the Institut national d’excellence en santé et en services sociaux (INESSS) issued a final negative recommendation for vamorolone (AGAMREE), meaning it is not recommended for public reimbursement in Québec for patients aged four and older with Duchenne muscular dystrophy.

Defeat Duchenne Canada Participates in CORD Conference in Toronto

On April 29–30, we attended the Rare Disease Day Conference hosted by the Canadian Organization for Rare Disorders (CORD) in Toronto. The event focused on Canada’s Rare Disease Strategy, accessing treatments, and strengthening the health system.

During the conference, Defeat Duchenne Canada met with representatives from Canada’s Drug Agency to discuss access to Duchenne treatments. We also engaged with Health Canada to highlight the importance of the Special Access Program (SAP), which can provide individuals living with Duchenne access to promising therapies before they receive full regulatory approval or public funding.