Cognitive problems are common in children with SMA, even if they are treated early on

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German clinicians looked at the frequency of cognitive impairment in a cohort of children with SMN1-related spinal muscular atrophy (SMA) type 1 who had received early treatment with one of the three innovative therapies available on the market:

  • 20 children were included in the study, 19 of whom were symptomatic (up to 3 copies of the SMN2 gene) and one presymptomatic,
  • 16 of them benefited from switches between these medications,
  • the investigators used the Bayley scales for the youngest children and the WPSII-IV from the age of 5,
  • 55% of the children had cognitive problems, especially the boys,
  • the severity of motor impairment and the need for ventilation and/or nutritional assistance were correlated with the frequency of these disorders.

These data will enable clinicians to better guide their therapeutic indications.

 

Cognitive function in SMA patients with 2 or 3 SMN2 copies treated with SMN-modifying or gene addition therapy during the first year of life. Steffens P, Weiss D, Perez A et al. Eur J Paediatr Neurol. 2024 Jul;51:17-23.

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