won’t stop while the provincial reimbursement process continues. We hope that provinces move swiftly to add Zolgensma to their drug plans. Access to Zolgensma will offer children with SMA the possibility of disease stabilization, drastically changing the outcome of this disease.”
Every year, roughly one in 10,000 newborns in Canada are born with SMA disorder, which is a rare genetic neuromuscular disorder caused by a faulty or absent SMN1 gene2. Newborns suffering from SMA lack a functioning SMN1 gene and lose motor neurons that control muscular activities such as breathing, eating, speaking, and walking. Muscles gradually deteriorate in strength if not treated on time. In the most serious cases, this finally results in paralysis, and in over 90% of cases, it results in permanent ventilation or death at age 2. Hence, it is critical to identify SMA and initiate therapy immediately, including proactive supportive care, to arrest permanent motor neuron loss and disease progression.
