Spinal muscular atrophy (SMA) is a genetic, progressive, and often terminal rare disease that affects an individual’s ability to walk, eat, and, ultimately, breathe. Debilitating and often fatal, SMA affects approximately one in 10,000 individuals and is a leading genetic cause of death among infants.
SMA impacts individuals across a range of ages – from infants and children to teens and adults – with varying levels of severity. Newborns and infants are most likely to develop infantile-onset SMA, the most severe form of the disease, which can lead to paralysis and prevent infants from performing the basic functions of life, such as swallowing or holding up their heads. Later-onset SMA is most common among teens and adults, who may experience significant muscle weakness and disability, such as the inability to stand or walk independently.
Individuals with two copies of the SMN2 gene, the gene responsible for making the survival motor neuron (SMN) protein, are most likely to develop infantile-onset SMA (also known as Type 1), while those with three or four copies are most likely to develop later-onset SMA (Types 2 and 3).
Therapy for SMA
Biogen is deeply committed to helping improve the lives of people with SMA, working closely with the SMA patient community with the goal of understanding and incorporating the perspectives of patients and families into the decisions we make. SPINRAZA™ (nusinersen) is approved by Health Canada, and Biogen and pCPA have reached an agreement to ensure SMA patients in Canada have access to treatment.
Once you have been prescribed with SPINRAZA™, your physician can refer you to the SMA 360◦™ Canada program, which provides access to a Family Access Manager (FAM). Your FAM will help guide you through the process and coordinate all aspects of treatment with SPINRAZA™, including:
- Insurance and financial support
- Drug logistics
- Travel assistance
- Product education
Learn more about our treatment for spinal muscular atrophy in Canada.