SMA impacts individuals across a range of ages — from infants and children to teens and adults — with varying levels of severity.1 Newborns and infants can develop infantile-onset SMA, the most severe form of the disease, which may lead to paralysis and prevent infants from performing the basic functions of life, such as swallowing or holding up their heads.2,3 Later-onset SMA is more common among children, teens, and adults, who may experience significant muscle weakness and disability, such as the inability to stand or walk independently.1,2,4
Previously, individuals with SMA and their families had no treatment options. This changed in December 2016 when Biogen received U.S. Food and Drug Administration (FDA) approval for the first therapy to treat SMA.5 The therapy has since been approved in more than 60 countries and over 14,000 living with SMA have been able to access treatment with the therapy.6
Biogen’s commitment to the SMA community is unwavering, and we continue to advance leading research aimed at addressing unmet needs and improving clinical outcomes for individuals impacted by the disease.
Biogen is collaborating with Ionis Pharmaceuticals to identify new therapeutic options — specifically, new antisense oligonucleotide (ASO) candidates.
We are also leveraging our learnings in SMA to advance potential therapeutic options and solutions in other neuromuscular indications with high unmet need.
Matilda is a busy four-year-old. Like most children of her age, she wants to do everything herself but due to living with Spinal Muscular Atrophy (SMA) sometimes Matilda needs a little extra help.
Tristram was around 18 months old when his parents Lorna and Ian noticed that he was not meeting his physical milestones. Activities like walking at the same pace as others became challenging, and he began to stumble and fall over.
Around 1 in every 40–50 people globally are carriers of the gene that causes SMA.7
SMA affects approximately one in every 10,000 births worldwide2,8,9,10 and in Australia.
Before treatment was available, SMA was the number one genetic cause of death of babies under two years11 in Australia.