Respiratory outcome of spinal muscular atrophy type 1 patients treated with nusinersen.

Almala Pınar ErgenekonCansu Yilmaz YegitMuruvvet CenkYasemin GokdemirEla Erdem EralpGülten ÖztürkOlcay UnverOzge Kenis CoskunEvrim Karadag SaygiDilsad TurkdoganBulent Karadag

Published in: Pediatrics international : official journal of the Japan Pediatric Society (2022)

Early initiation of nusinersen treatment in SMA1 patients may alter the disease's natural course.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
peritoneal dialysis
patient reported outcomes
combination therapy
smoking cessation
patient reported