Nusinersen for spinal muscular atrophy type 1: Real-world respiratory experience.

Moran Lavie Nir Diamant Michal CahalEfraim SadotMoria Be'erAviva Fattal-ValevskiLiora SagiKeren A DomanyIsrael Amirav

Published in: Pediatric pulmonology (2020)

Most of our patients were stable in their need for assisted ventilation and did not worsen as expected in SMA1, nor did they improve as might be hoped. Future studies are needed to determine if earlier treatment with Nusinersen might result in respiratory outcomes superior to those reported in this real-life study.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
current status
respiratory tract
patient reported outcomes
intensive care unit
weight loss
respiratory failure
case control