Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment.

Eugenio MercuriGiovanni BaranelloOdile Boespflug-TanguyLiesbeth De WaeleNathalie GoemansJanbernd KirschnerRiccardo MassonElena S MazzoneAstrid PechmannMaria Carmela PeraCarole VuillerotSilvia Bader-WederMarianne GerberKsenija GorniJanine HoffartHeidemarie KletzlCarmen MartinTammy McIverRenata S ScalcoWai Yin YeungLaurent Servaisnull null

Published in: European journal of neurology (2022)

SUNFISH Part 1 demonstrated a twofold increase in SMN protein after treatment with risdiplam. The observed safety profile supported the initiation of the pivotal Part 2 study. The long-term efficacy and safety of risdiplam are being assessed with ongoing treatment.

Keyphrases

study protocol
clinical trial
placebo controlled
double blind
squamous cell carcinoma
phase ii
randomized controlled trial
small molecule
combination therapy
replacement therapy
protein protein
smoking cessation