SMA is a rare and debilitating autosomal recessive neuromuscular disease characterised by motor neuron degeneration and loss of muscle strength.1,2 Age of onset determines the level of motor neuron degeneration.1,2
Watch the video to learn how the disease works or visit page below.
The natural progression of SMA involves irreversible loss of motor function,3 which makes it important to confirm diagnosis as soon as possible. The sooner a child is diagnosed, the sooner care can start, and outcomes can be improved.4
Eventually your adult SMA patients may not be able to scroll down this page. SMA is a debilitating neuromuscular disease characterised by motor neuron degeneration and loss of muscle strength.1 The loss of basic motor functions will require appropriate intervention.
The natural progression of SMA involves loss of motor function.3 There are several scales such as CHOP INTEND, and HINE to help measure disease progression of SMA, and tools to explain what the disease entails, which could be useful in your practice.
The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families.
Photographs are for illustrative purposes only.
Keep an eye out for what is happening in the world of SMA events. All over the world events take place discussing the latest advances in SMA and SMA care, which could be useful in your practice.
Together in SMA was created in support of that unity, and with the goal of becoming a valued tool for members of the team. Here you’ll find clinical information and care strategies that some people use to help manage the disease.
Biogen is building on our legacy of rare disease research and development to explore potential treatment options. And even then, we’ll still be together in SMA.
1. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet 2008;371(9630):2120-2133.
2. Darras BT, Royden Jones H Jr, Ryan MM, De Vivo DC, eds. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015.
3. Swoboda KJ, et al. Perspectives on clinical Trials in Spinal Muscular Atrophy. J Child Neurol 2007;22(8):957-966.
4. Rothwell E, Anderson RA, Swoboda KJ, Stark L, Botkin JR. Public attitudes regarding a pilot study of newborn screening for spinal muscular atrophy. Am J Med Genet A 2013;161A(4):679-686.