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.
