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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