A useful electrophysiological score in gene therapy for SMA

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French clinicians report their experience with measuring compound muscle action potentials (CMAP) in infants with symptomatic SMA who have received gene therapy (onasemnogene abeparvovec):

  • 19 infants with SMA, including 12 with two copies of the SMN2 gene, were included in the study,
  • their CMAPs were measured at several nerves (median, ulnar, peroneal and tibial) at several points in time,
  • a cumulative motor index (CMI) was constructed from the values obtained,
  • after administration of gene therapy, this index improved significantly and then stabilised at relatively low values after 24 months,
  • the baseline CMI (before therapy) proved to be fairly predictive of the benefit of treatment.

This minimally invasive technique is a very useful aid in initiating treatment and for prognosis.

 

Cumulative motor index in spinal muscular atrophy after gene therapy: baseline predicts maximal recovery. Barrois R, Barnerias C, Hervé A et al. Clin Neurophysiol. 2025 Aug.

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