Nusinersen (Spinraza®) can effectively treat infants with spinal muscular atrophy (SMA) and is associated with fewer severe adverse events, according to a systematic review and meta-analysis of randomized controlled trials. 

To assess the beneficial and adverse effects of nusinersen, a team led by James Robert Brašić, MD, MPH, of Johns Hopkins University School of Medicine in Baltimore, Maryland, used Preferred Reporting for Systematic Review and Meta-Analysis (PRISMA) to systematically search for and meta-analyze published randomized control trials of nusinersen for SMA up until July 22, 2021.

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They identified 3 clinical trials that satisfied the inclusion and exclusion criteria. The trials included a total of 274 SMA patients. Of these, 178 patients received nusinersen while the rest were in the control group. 

The results showed that there were significant risk differences in motor milestone response and improvement in the Hammersmith Infant Neurological Examination (HINE-2) score between patients treated with nusinersen and controls.

In addition, there was a significant decrease in the risk ratio for severe adverse events with nusinersen treatment. Any adverse event that resulted in treatment discontinuation also decreased significantly. There was a similar trend in terms of all adverse events and serious adverse events, but this was not statistically significant.

“This review provides evidence that nusinersen treatment was effective in the treatment of infants with SMA and was associated with fewer severe adverse events when compared to the control group,” the researchers wrote in an article published in Medicina, adding that “the initial results for the safety and efficacy of nusinersen are promising.”

According to the authors of the study, more randomized clinical trials are necessary to establish this evidence.

Nusinersen is an antisense oligonucleotide that aims to increase the level of survival motor neuron protein synthesized from the SMN2 gene. It is approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of all types of SMA.


Said Abbas K, Eltaras MM, El-Shahat NA, Abdelazeem B, Shaqfeh M, Brašić JR. The safety and efficacy of nusinersen in the treatment of spinal muscular atrophy: a systematic review and meta-analysis of randomized controlled trials. Medicina. 2022;58(2):213. doi:10.3390/medicina58020213