Risdiplam in Type 1 Spinal Muscular Atrophy.

Giovanni BaranelloBasil T DarrasJohn W DayNicolas DeconinckAndrea KleinRiccardo MassonEugenio MercuriKristy RoseMuna El-KhairiMarianne GerberKsenija GorniOmar KhwajaHeidemarie KletzlRenata S ScalcoTimothy SeabrookPaulo FontouraLaurent Servaisnull null

Published in: The New England journal of medicine (2021)

In infants with type 1 spinal muscular atrophy, treatment with oral risdiplam led to an increased expression of functional SMN protein in the blood. (Funded by F. Hoffmann-La Roche; ClinicalTrials.gov number, NCT02913482.).

Keyphrases

poor prognosis
binding protein
protein protein
amino acid
smoking cessation
light emitting