Response of plasma microRNAs to nusinersen treatment in patients with SMA.

Irina T ZaharievaMariacristina ScotoKarolina Aragon-GawinskaDeborah RidoutBruno DoresteLaurent ServaisFrancesco MuntoniHaiyan Zhou

Published in: Annals of clinical and translational neurology (2022)

Blood microRNAs could be used as biomarkers to indicate SMA patients' response to nusinersen and to monitor the efficacy of the therapeutic intervention. In addition, some of these microRNAs provide insight into processes involved in SMA that could be exploited as novel therapeutic targets.

Keyphrases

end stage renal disease
ejection fraction
randomized controlled trial
newly diagnosed
chronic kidney disease
prognostic factors
peritoneal dialysis
patient reported outcomes
replacement therapy