Defeat Duchenne Canada, La Force DMD, and Muscular Dystrophy Canada are working together to conduct a joint national survey to understand the impact of Duchenne muscular dystrophy on individuals and their families.

The findings will help inform patient-group submissions to Canada’s Drug Agency (CDA-AMC) and Québec’s Institut national d’excellence en santé et en services sociaux (INESSS) about givinostat, a potential new treatment for Duchenne currently under Priority Review by Health Canada.

Why your voice matters

For people living with Duchenne, time is muscle. As new treatments move through the Canadian regulatory and reimbursement processes, it is important that decision-makers understand the real-life experiences, challenges, and needs of the Duchenne community.

Your experiences can help ensure that the perspectives of people living with Duchenne and their families are reflected in the evaluation of new treatment options.

What is the drug review process?

CDA-AMC reviews new treatments and makes recommendations to Canada’s provincial and territorial governments about whether they should be publicly funded, with the exception of Québec. In Québec, INESSS conducts a separate review and makes its own recommendation to the provincial government.

Patient and caregiver input helps inform these reviews by sharing what matters most to the people and families affected by a condition.

Before you begin

• Who can participate: The survey is open to people living with Duchenne in Canada and their parents, family members, or unpaid caregivers. People living with Duchenne are encouraged to answer directly when possible. A caregiver may provide support or answer on behalf of someone else, but should indicate whose perspective is being represented.
• Voluntary participation: Taking part is voluntary. You may skip any question and stop at any time.
• Time required: The survey takes approximately 15–20 minutes. You may save your responses and return later.
• Privacy: Responses will be summarized and analyzed. De-identified quotations may be used in patient-group submissions or related public reports. Names and contact details will not be included with survey responses.
• Follow-up interviews: If you wish to volunteer for a follow-up interview, you will be directed to a separate contact form after submitting the survey. Your contact information will not be stored with your survey responses.

If you have questions about the survey, please contact:

Rosalind O’Connell
National Director, Advocacy
Defeat Duchenne Canada
[email protected]

Thank you for taking the time to share your voice and contribute to this important work.