Spinal cord epidural stimulation proves effective in type III SMA

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An initial study evaluated epidural stimulation of the spinal cord in three patients with type III spinal muscular atrophy (SMA). While motor deficits may persist with Spinraza®, Zolgensma® and Evrysdi®, this technique could directly target the dysfunction of the neuronal circuitry of the spinal cord involved in the progression of the disease.

  • The three participants had weakness in their lower limbs and were able to stand and walk.
  • Electrodes were placed temporarily in the lumbosacral region on either side of the spinal cord and the spinal cord was stimulated for two hours a day for one month.
  • Muscular strength, walking quality and endurance were improved in the various tests.
  • All three participants improved the distance covered in the six-minute walking test by at least 20 m.
  • Electrophysiological data showed that stimulation improved motor neuron function, with effects occurring during both active and inactive periods of stimulation.
  • No adverse events were reported.

 

First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy. Prat-Ortega G, Ensel S, Donadio S et al. Nat Med. 2025 Feb.

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