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Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)

  • Maryam Oskoui*
  • , John W Day
  • , Nicolas Deconinck
  • , Elena S Mazzone
  • , Andres Nascimento
  • , Kayoko Saito
  • , Carole Vuillerot
  • , Giovanni Baranello
  • , Nathalie Goemans
  • , Janbernd Kirschner
  • , Anna Kostera-Pruszczyk
  • , Laurent Servais
  • , Gergely Papp
  • , Ksenija Gorni
  • , Heidemarie Kletzl
  • , Carmen Martin
  • , Tammy McIver
  • , Renata S Scalco
  • , Hannah Staunton
  • , Wai Yin Yeung
  • Paulo Fontoura, Eugenio Maria Mercuri
*Corresponding author
  • McGill University
  • Ghent University
  • Queen Fabiola Children's University Hospital
  • SJD Barcelona Children's Hospital
  • Tokyo Women's Medical University
  • Hospices Civils de Lyon-Centre Léon Bérard
  • Université de Lyon
  • Great Ormond Street Hospital for Children
  • IRCCS Fondazione Istituto Neurologico Carlo Besta - Milano
  • KU Leuven
  • University of Freiburg
  • Medical University of Warsaw
  • University of Oxford
  • Hôpital Armand Trousseau
  • University of Liege
  • F. Hoffmann-La Roche AG
  • Roche Products Limited

Research output: Contribution to journalArticle

Abstract

The original version of this article unfortunately contained a mistake. The corrected details are given below for your reading. In figure 1, there is an error in the n numbers below the graph in Panel 1b for the placebo group. The n numbers underneath Panel 1b should be 58 58 50. There is an error within Fig. 4. The dashed lines at ~ − 1.4 should be at 0. They have been moved downwards and are no longer in the correct place. The corrected Figs. 1 and 4 are given in the following page. (Figure presented.) (Figure presented.) Change from baseline in MFM32 total score in patients treated with risdiplam for up to 24 months and those who previously received placebo until study month 12. aThirty-one percent (55/180) of the SUNFISH intent-to-treat population were 2–5 years old at baseline. b± 95% CI. cBaseline is the last measurement prior to the first dose of risdiplam or placebo. dData cut-off: 30 Sep 2020. eData cut-off: 6 Sep 2019. fPatients in the placebo arm received placebo for 12 months followed by risdiplam treatment for 12 months. gNumber of patients with valid results = number of patients with an available total score (result) at respective time points. Intent-to-treat patients. hPatients in the placebo arm received placebo for 12 months followed by risdiplam treatment for 12 months. Placebo period not shown in this graph. CI confidence interval, MFM32 32-item motor function measure Change in caregiver- and patient-reported SMAIS upper limb total score from baseline in patients receiving risdiplam for up to 24 months and those who previously received placebo up to study month 12. a± 95% CI. Baseline is the last measurement prior to the first dose of risdiplam or placebo. bData cut-off: 30 Sep 2020. cData cut-off: 6 Sep 2019. dPatients in the placebo arm received placebo for 12 months followed by risdiplam treatment for 12 months. Risdiplam period not shown in this graph. eNumber of patients with valid results = number of patients with an available total score (result) at respective time points. Intent-to-treat patients. SMAIS scores range from 0 to 44 following rescoring to a 0–2 response scale for each item. Higher scores indicate greater independence in completing daily activities. CI confidence interval, SMA spinal muscular atrophy, SMAIS SMA Independence Scale, SMAIS-ULM SMA Independence Scale-Upper Limb Module.
Original languageEnglish
Pages (from-to)2547-2549
Number of pages3
JournalJournal of Neurology
Volume270
Issue number5
DOIs
Publication statusPublished - 2023

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

  1. SDG 3 - Good Health and Well-being
    SDG 3 Good Health and Well-being

All Science Journal Classification (ASJC) codes

  • Neurology
  • Clinical Neurology

Keywords

  • risdiplam
  • spinal muscular atrophy (SMA)

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