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

Maryam Oskoui¹, John W Day², Nicolas Deconinck^{3

4}, Elena S Mazzone⁵, Andres Nascimento⁶, Kayoko Saito⁷, Carole Vuillerot^{8

9}, Giovanni Baranello^{10

11}, Nathalie Goemans¹², Janbernd Kirschner¹³, Anna Kostera-Pruszczyk¹⁴, Laurent Servais^{15

16

17}, Gergely Papp¹⁸, Ksenija Gorni¹⁹, Heidemarie Kletzl²⁰, Carmen Martin²¹, Tammy McIver²¹, Renata S Scalco²², Hannah Staunton²¹, Wai Yin Yeung²¹, Paulo Fontoura¹⁹, Eugenio Mercuri⁵; SUNFISH Working Group

Affiliations

¹ Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca.
² Department of Neurology, Stanford University, Palo Alto, CA, USA.
³ Neuromuscular Reference Center, UZ Gent, Ghent, Belgium.
⁴ Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium.
⁵ Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
⁶ Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain.
⁷ Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
⁸ Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.
⁹ Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France.
¹⁰ 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.
¹¹ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
¹² Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium.
¹³ Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.
¹⁴ Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
¹⁵ I-Motion-Hôpital Armand Trousseau, Paris, France.
¹⁶ MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.
¹⁷ Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
¹⁸ Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
¹⁹ PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
²⁰ Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
²¹ Roche Products Ltd, Welwyn Garden City, UK.
²² Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.

PMID: 37071150
PMCID: PMC10129951
DOI: 10.1007/s00415-023-11658-6

Published Erratum

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 et al. J Neurol. 2023 May.

. 2023 May;270(5):2547-2549.

doi: 10.1007/s00415-023-11658-6.

Authors

Affiliations

¹ Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca.
² Department of Neurology, Stanford University, Palo Alto, CA, USA.
³ Neuromuscular Reference Center, UZ Gent, Ghent, Belgium.
⁴ Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium.
⁵ Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
⁶ Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain.
⁷ Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
⁸ Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.
⁹ Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France.
¹⁰ 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.
¹¹ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
¹² Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium.
¹³ Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.
¹⁴ Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
¹⁵ I-Motion-Hôpital Armand Trousseau, Paris, France.
¹⁶ MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.
¹⁷ Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
¹⁸ Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
¹⁹ PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
²⁰ Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
²¹ Roche Products Ltd, Welwyn Garden City, UK.
²² Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.

PMID: 37071150
PMCID: PMC10129951
DOI: 10.1007/s00415-023-11658-6

No abstract available

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Figures

**Fig. 1**
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

**Fig. 4**
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

See this image and copyright information in PMC

Erratum for

Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).
Oskoui M, Day JW, Deconinck N, Mazzone ES, Nascimento A, Saito K, Vuillerot C, Baranello G, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Papp G, Gorni K, Kletzl H, Martin C, McIver T, Scalco RS, Staunton H, Yeung WY, Fontoura P, Mercuri E; SUNFISH Working Group. Oskoui M, et al. J Neurol. 2023 May;270(5):2531-2546. doi: 10.1007/s00415-023-11560-1. Epub 2023 Feb 3. J Neurol. 2023. PMID: 36735057 Free PMC article. Clinical Trial.

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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)

Affiliations

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

Authors

Affiliations

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Erratum for

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