Key result
Nusinersen for spinal muscular atrophy sees ~31% treatment interruption over 30 months.
Why the study?
The authors sought to report clinical outcomes of nusinersen in patients with SMA type 1 or 2 and comment on the ethical implications of this treatment.
Does nusinersen improve clinical outcomes in children with SMA type 1 or 2?
Does nusinersen improve clinical outcomes in children with SMA type 1 or 2?
Answers are grounded in this paper and cite their sources.
In a cohort of 35 children with SMA type 1 or 2, nusinersen showed mixed efficacy and tolerability, highlighting the need for careful patient selection and weighing of benefits against treatment discomfort.
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Agosto et al. (2021) conducted a letter in Spinal muscular atrophy type 1 or 2 (n=35). Nusinersen was evaluated on Treatment response and interruption rates. Among 35 children with spinal muscular atrophy type 1 or 2 treated with nusinersen, 11 required treatment interruption over 30 months due to adverse events, lack of efficacy, or family choice.
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