Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.

Maria Carmela PeraGiorgia Coratti Francesca BovisMarika PaneAmy PasternakJacqueline MontesValeria A SansoneSally Dunaway YoungTina DuongSonia MessinaIrene MizzoniAdele D'AmicoMatthew CivitelloAllan M GlanzmanClaudio BrunoFrancesca SalminSimone MorandoRoberto De SanctisMaria SframeliLaura AntonaciAnna Lia FrongiaAnnemarie RohwerMariacristina ScotoDarryl C De VivoBasil T DarrasJohn DayWilliam MartensKatia A PatanellaEnrico BertiniFrancesco MuntoniRichard FinkelEugenio Mercurinull null

Published in: Annals of clinical and translational neurology (2021)

Our results expand the available data on the effect of Nusinersen on type III patients, so far mostly limited to data from adult type III patients.

Keyphrases

type iii
end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
prognostic factors
electronic health record
patient reported outcomes
big data