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 nullPublished 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.