Introduction

Spinal muscular atrophy (SMA) is one of the leading genetic causes of death in infants¹

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SMA is a rare neuromuscular disease caused by the degeneration of motor neurons in the spinal cord and brainstem. SMA is characterised by skeletal muscle atrophy and weakness.^2,3 For the purposes of clinical classification, individuals with SMA are sometimes described in terms of types based on age of onset, severity of symptoms, and disease progression.^2,4

SMA causes progressive degeneration of motor neurons in the spinal cord, leading to muscle atrophy and weakness.²

Learn more about the genetic cause of SMA, including the roles of the SMN1 and SMN2 genes in individuals with SMA, and discover how this disease can present with a wide range of symptoms and clinical challenges.

What causes SMA?

Find out more about the genetic cause of SMA.

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How does untreated SMA manifest?

Learn more about the natural history of untreated SMA.

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References

Butchbach ME. Copy number variations in the survival motor neuron genes: implications for spinal muscular atrophy and other neurodegenerative diseases. Front Mol Biosci. 2016;3:7.
Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.
Wang CH, Finkel RS, Bertini ES, et al; and Participants of the International Conference on SMA Standard of Care. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027-1049.
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.
Jones C, Zielinski D, Vinikoor L, Farwell W; Biogen Idec, Safety and Benefit Risk Management; Cambridge, MA; McGill University, Montreal, Quebec; Biogen Idec, Clinical Development, Cambridge, MA. Systematic review of incidence and prevalence of spinal muscular atrophy (SMA). Poster presented at: 11th European Paediatric Neurology Society Congress; May 27-30, 2015; Vienna, Austria.
Pearn J. Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular dystrophy. J Med Genet. 1978;15(6):409-413.
Cure SMA. Genetics. https://www.curesma.org/genetics/ . Last Accessed 4 February 2022.

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Biogen-176301-HKG-09/2022

Introduction

Spinal muscular atrophy (SMA) is one of the leading genetic causes of death in infants1

Spinal muscular atrophy (SMA) is one of the leading genetic causes of death in infants¹