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.

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Promote SMN2 expression to increase SMN protein production1-3

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Replace the missing or defective SMN1 gene1-4

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Modify SMN2 expression to increase the amount of functional SMN protein produced1,4

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Substitute motor neurone with stem cells, aiming to enhance muscle strength1, 6

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Stabilise/protect any SMN protein produced1, 5

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Protect motor neurons from damage despite SMN protein deficiency1-3

References
  1. Iascone DM et al. F1000Prime Rep 2015; 7: 04.
  2. Darras BT. Pediatr Clin North Am 2015; 62: 743–66.
  3. Van Meerbeke JP and Sumner CJ. Discov Med 2011; 12: 291–305.
  4. Faravelli I et al. Nat Rev Neurol 2015; 11: 351–9.
  5. Lunn MR et al. Chem Biol 2004; 11: 1489–93.
  6. Shin S et al. Stem Cells Dev 2005; 14: 266–9.