[en] 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.
Disciplines :
Pediatrics
Author, co-author :
Oskoui, Maryam ; Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca
Day, John W ; Department of Neurology, Stanford University, Palo Alto, CA, USA
Deconinck, Nicolas ; Neuromuscular Reference Center, UZ Gent, Ghent, Belgium ; Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium
Mazzone, Elena S ; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy
Nascimento, Andres ; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain
Saito, Kayoko ; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan
Vuillerot, Carole ; Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France ; Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France
Baranello, Giovanni ; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London and Great Ormond Street Hospital Trust, London, UK ; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
Goemans, Nathalie ; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium
Kirschner, Janbernd ; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany
Kostera-Pruszczyk, Anna ; Department of Neurology, Medical University of Warsaw, Warsaw, Poland
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques ; I-Motion-Hôpital Armand Trousseau, Paris, France ; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK
Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA).
Alternative titles :
[en] Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA) (Journal of Neurology, (2023), 270, 5, (2531-2546), 10.1007/s00415-023-11560-1)
