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Drug Evaluation
Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)
Theodora Markatia MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK;b Department of Paediatric Neurology, Oxford University Hospitals NHS Foundation Trust, Oxford, UKhttps://orcid.org/0000-0003-0115-264XView further author information
, Gemma Fishera MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK;b Department of Paediatric Neurology, Oxford University Hospitals NHS Foundation Trust, Oxford, UKhttps://orcid.org/0000-0001-5351-7327View further author information
, Sithara Ramdasa MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK;b Department of Paediatric Neurology, Oxford University Hospitals NHS Foundation Trust, Oxford, UKhttps://orcid.org/0000-0003-3790-2190View further author information
& Laurent Servaisa MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK;b Department of Paediatric Neurology, Oxford University Hospitals NHS Foundation Trust, Oxford, UK;c Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liege, BelgiumCorrespondence[email protected]
https://orcid.org/0000-0001-9270-4061View further author information
https://orcid.org/0000-0001-9270-4061View further author information
Pages 451-461
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Received 17 Dec 2021, Accepted 20 Mar 2022, Published online: 11 Apr 2022
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