A new study found no correlation between delayed treatment due to the COVID-19 pandemic lockdown and a change in functional scores in children and adolescents with spinal muscular atrophy (SMA) in Italy.

Due to the lockdown, 8 out of 25 patients (32%) experienced delays (median, 58 days; range 26-91) in their infusions of nusinersen, but only marginal changes in the functional scores were reported in 7 of these patients.

The study, published in Frontiers in Neurology, found only 3 patients (12%) had a decrease in functional scores greater than 2 points at short-period evaluation (2 months after the end of the first lockdown) as well as in long-term evaluation (the following months).

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A total of 9 patients showed reductions in their functional scores at the first follow-up after the lockdown, but only 3 had decreases of more than 2 points. These 3 patients had strong clinical reasons for the decline including 1 patient with poor compliance to the standard of care due to a poor social context (patient number 6), 1 with a more severe disease trajectory even before the lockdown (patient number 21), and an adolescent whose scoliosis had worsened in the months directly prior to the lockdown (patient number 13).

In the long-term, 10 patients reported worsening of symptoms but only 3 had relevant (>-2) changes in functional scores. One of the patients was an adolescent with scoliosis who did not improve from the first follow-up (patient number 13). The other 2 patients experienced their decline between the 2 follow-up visits which appeared to be due to social situations with one being cared for by a fatigued and single parent, and the other by parents depressed from the loss of their jobs.

Despite some patients showing decreases in functional scores, 5 patients (20%) showed improvements at their first visit after lockdown. At the second visit after the lockdown, 8 patients (32%) showed an improvement over their values before lockdown, with 1 patient experiencing a 7-point increase in their functional scores due to the presence of 2 caregivers during the lockdown period.

Both patients numbered 6 and 21, who had functional score deficits greater than -2 points at the first visit after lockdown, increased their functional scores by the second visit.

“[The importance of home therapy] is confirmed by the fact that children who had both parents caring for them and helping each other during the lockdown showed the best improvement in motor function, while in the presence of social issues, above all in single-parent families, children suffered a lack of home institutional help,” the authors concluded.


Agosto C, Salamon E, Giacomelli L, Papa S, Benedetti F, Benini F. Effect of the COVID-19 pandemic on children with SMA receiving nusinersen: what is missed and what is gained? Front Neurol. 2021;12:704928. doi:10.3389/fneur.2021.704928