Care guidelines

Standards of care in SMA

Management of SMA has changed extensively in recent years, with research and development of new therapies and the evolution of care guidelines.

Changing landscape of SMA care

Until recently, care in SMA was focussed on physical interventions and managing complications, but with new treatments available and emerging, the way SMA is managed is undergoing dynamic change.

Promote SMN2 expression to increase SMN protein production^1-3

Replace the missing or defective SMN1 gene^1-4

Modify SMN2 expression to increase the amount of functional SMN protein produced^1,4

Substitute motor neurone with stem cells, aiming to enhance muscle strength^{1, 6}

Stabilise/protect any SMN protein produced^{1, 5}

Protect motor neurons from damage despite SMN protein deficiency^1-3

References

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Van Meerbeke JP and Sumner CJ. Discov Med 2011; 12: 291–305.
Faravelli I et al. Nat Rev Neurol 2015; 11: 351–9.
Lunn MR et al. Chem Biol 2004; 11: 1489–93.
Shin S et al. Stem Cells Dev 2005; 14: 266–9.