Féline E V Scheijmans, Inge Cuppen, Ruben P A van Eijk, Camiel A Wijngaarde, Marja A G C Schoenmakers, Danny R van der Woude, Bart Bartels, Esther S Veldhoen, Irene L B Oude Lansink, Ewout J N Groen, Fay-Lynn Asselman, Renske I Wadman, W Ludo van der Pol
Nusinersen (Spinraza®) improves survival of infants with hereditary proximal spinal muscular atrophy and motor function in children up to 12 years. Population-based assessments of treatment efficacy are limited and confined to select cohorts of patients. We performed a nationwide, population-based, single-centre cohort study in children with spinal muscular atrophy younger than 9.5 years at start of treatment in line with reimbursement criteria in the Netherlands. We assessed age-relevant motor function scores, the need for tube feeding, hours of ventilatory support and documented adverse events...
2022: Brain communications