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Felix Kleefeld

Ruhr University of Bochum

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Scholarly Papers (1)

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Real-World Outcomes of Risdiplam in 5q Spinal Muscular Atrophy Over 30 Months in Adolescents and Adults: A Multicentre, Prospective, Observational SMArtCARE Study

Number of pages: 28 Posted: 05 Feb 2026
Ludwig Maximilian University of Munich (LMU), Heidelberg University, University of Duisburg-Essen, University of Duisburg-Essen, Medical University of Innsbruck, Universitätsklinikum Leipzig, Ludwig Maximilian University of Munich (LMU), Medical University of Vienna, Technische Universität München (TUM), Johannes Kepler University Linz, Dresden University of Technology, Justus-Liebig-University, Ludwig Maximilian University of Munich (LMU), University of Göttingen, University of Bonn - University Hospital Bonn (UKB), Friedrich-Schiller-Universität Jena - University Hospital Jena, University of Hamburg - University Medical Center Hamburg-Eppendorf, Klinikum Bremen Mitte, Medical University of Vienna, University of Freiburg, Ruhr University of Bochum, University of Duisburg-Essen, Paracelsus Medical University Salzburg, University of Duisburg-Essen - Department of Pediatric Neurology, Medical University of Innsbruck, Martin Luther University of Halle-Wittenberg - University Hospital Halle (Saale), Universitätsklinikum Leipzig, Universitätsklinikum Münster, Kantonsspital St Gallen, University of Freiburg, Hannover Medical School, Ordensklinikum Linz Hospital Barmherzige Schwestern, University of Duisburg-Essen - University Hospital of Essen, Klinikum Stuttgart, Friedrich-Schiller-Universität Jena - University Hospital Jena, Heidelberg University, DKD HELIOS Hospital Wiesbaden, Klinikum Kassel, University of Münster - Department of Pediatric Neurology, Medical University of Graz, Klinikum Klagenfurt am Wörthersee, Universitätsklinikum Schleswig-Holstein, Friedrich-Alexander-Universität Erlangen-Nürnberg - Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander-Universität Erlangen-Nürnberg, Ulm University - Department of Neurology, Ludwig Maximilian University of Munich (LMU), University of Cologne - University Hospital of Cologne, University of Würzburg, Independent, University of Ottawa and University of Duisburg-Essen - Department of Neurology
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Abstract:

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Oral therapy, Motor Neuron Disease, Risdiplam, Small molecule, Spinal muscular atrophy