Spinal cord epidural stimulation proves effective in type III SMA

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.