January 4, 2022 – Muscular Dystrophy Canada (MDC) has submitted patient input to the Canadian Agency for Drugs and Technologies in Health (CADTH) in support of expanding access to nusinersen (Spinraza) for adults living with spinal muscular atrophy (SMA).
Spinal muscular atrophy is a severe, inherited, progressive neuromuscular disease that causes major problems with walking, muscle strength, fine motor skills, and the basic physical functions of breathing, swallowing, and feeding. Until recently, there were limited treatment options for SMA, but prognosis has been transformed with the recent availability of a number of effective disease-modifying therapies, including nusinersen.
The patient input submission aims to help address the significant treatment gap for adults affected by SMA. It captures the voices and lived experiences of adults in Canada’s SMA community, highlighting the disease’s significant impact related to their independence, daily activities, mental health and well-being, energy levels and work participation.
MDC continues to advocate for equitable access to innovative treatments that improve health outcomes and enhance quality of life for Canadians affected by neuromuscular disorders.
