Cognitive profile and treatment in SMA type I

A study reveals for the first time the effects of three available treatments for SMA on the cognitive profile of infants with SMA type I:

  • 18 infants participated in the study, 11 SMA type I treated post-symptomatically and 7 presymptomatically,
  • 11 received Spinraza, three received Evrysdi and four received Zolgensma,
  • In the presymptomatically treated infants, the cognitive score was mean, and for one of them, lower than the mean,
  • the cognitive score of those treated post-symptomatically was rather below the mean, with a tendency to improve during the three years of follow-up.

The authors conclude that the cognitive profile should also be taken into account when treating patients with type I SMA.


Longitudinal developmental profile of newborns and toddlers treated for spinal muscular atrophy. Ngawa M, Dal Farra F, Marinescu AD, Servais L. Ther Adv Neurol Disord. 2023 Fév.