Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo‐controlled, dose‐finding trial followed by 24 months of treatment
European Journal of Neurology2022Vol. 30(7), pp. 1945–1956
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Eugenio Mercuri, Giovanni Baranello, Odile Boespflug‐Tanguy, Liesbeth De Waele, Nathalie Goemans, Janbernd Kirschner, Riccardo Masson, Elena Mazzone, Astrid Pechmann, Maria Carmela Pera, Carole Vuillerot, Silvia Bader‐Weder, Marianne Gerber, Ksenija Gorni, Janine Hoffart, Heidemarie Kletzl, Carmen Martín, Tammy McIver, R. Scalco, Wai Yin Yeung, Laurent Servais, SUNFISH Working Group
Abstract
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.
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