October 5, 2021 – Muscular Dystrophy Canada (MDC) has submitted patient input to the Canadian Agency for Drugs and Technologies in Health (CADTH) in support of public reimbursement for Nexviazyme® (avalglucosidase alfa-ngpt), a treatment for individuals living with Pompe disease.
Approximately 60 people in Canada are living with Pompe disease, which can result in cardiomyopathy, progressive muscle weakness, respiratory failure, and heart failure.
The submission draws on the lived experiences of Canadians affected by Pompe disease and shares how living with Pompe disease affects people’s day-to-day lives, with themes including its impact on mobility, strength, balance and energy; breathing; and mental health as well as affecting the ability to participate in daily activities and having a negative impact on the family. It also shares the experiences of those in the Pompe community who have had experience with the therapy under review.
This submission reflects MDC’s continued commitment to ensuring equitable access to new therapies for all Canadians affected by neuromuscular disorders. By amplifying patient voices in Canada’s drug review processes, MDC is helping to shape a more responsive and inclusive healthcare system.
Read the submission now (available in English only)
