. 2023;10(1):29-40.

doi: 10.3233/JND-221600.

Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study

Astrid Pechmann¹, Max Behrens², Katharina Dörnbrack³, Adrian Tassoni³, Franziska Wenzel³, Sabine Stein¹, Sibylle Vogt¹, Daniela Zöller², Günther Bernert⁴, Tim Hagenacker⁵, Ulrike Schara-Schmidt⁶, Maggie C Walter⁷, Meike Steinbach⁸, Astrid Blaschek⁹, Matthias Baumann¹⁰, Manuela Baumgartner¹¹, Benedikt Becker¹², Marina Flotats-Bastardas¹³, Johannes Friese¹⁴, Rene Günther¹⁵, Andreas Hahn¹⁶, Hanna Küpper¹⁷, Jessika Johannsen¹⁸, Christoph Kamm¹⁹, Jan Christoph Koch²⁰, Cornelia Köhler²¹, Heike Kölbel⁶, Kirsten Kolzter²², Arpad von Moers²³, Steffen Naegel²⁴, Christoph Neuwirth²⁵, Susanne Petri²⁶, Annekathrin Rödiger²⁷, Mareike Schimmel²⁸, Bertold Schrank²⁹, Gudrun Schreiber³⁰, Martin Smitka³¹, Christian Stadler³², Elisabeth Steiner³³, Eva Stögmann³⁴, Regina Trollmann³⁵, Matthias Türk³⁶, Markus Weiler³⁷, Corinna Stoltenburg³⁸, Ekkehard Willichowsky³⁹, Daniel Zeller⁴⁰, Andreas Ziegler⁴¹, Hanns Lochmüller^{1

42

43

44}, Janbernd Kirschner¹; SMArtCARE Study Group

Collaborators, Affiliations

Collaborators

SMArtCARE Study Group:
Lisa Ameshofer, Barbara Andres, Daniela Angelova-Toshkina, Daniela Banholzer, Christina Bant, Petra Baum, Sandra Baumann, Ute Baur, Bettina Behring, Julia Bellut, Astrid Bertsche, Andrea Bevot, Jasmin Bischofberger, Lisa Bitzan, Bogdan Bjelica, Markus Blankenburg, Sandra Böger, Friederike Bonetti, Anke Bongartz, Svenja Brakemeier, Lisa Bratka, Nathalie Braun, Sarah Braun, Brigitte Brauner, Christa Bretschneider, Nadine Burgenmeister, Bea Burke, Sebahattin Cirak, Isabell Cordts, Andrea Dall, Heike de Vries, Adela Della Marina, Jonas Denecke, Marcus Deschauer, Zylfie Dibrani, Uta Diebold, Lutz Dondit, Jessica Drebes, Joenna Driemeyer, Vladimir Dukic, Matthias Eckenweiler, Astrid Eisenkölbl, Mirjam Eminger, Michal Fischer, Cornelia Fischer, Maren Freigang, Philippa Gaiser, Andrea Gangfuß, Gehrke Tasch, Stephanie Geitmann, Annette George, Magdalena Gosk-Tomek, Susanne Grinzinger, Kristina Gröning, Martin Groß, Anne-Katrin Güttsches, Anna Hagenmeyer, Hans Hartmann, Julia Haverkamp, Miriam Hiebeler, Annegret Hoevel, Georg Friedrich Hoffmann, Britta Holtkamp, Dorothea Holzwarth, Annette Homma, Veronka Horber, Viola Horneff, Carolin Hörnig, Anna Hotter, Andrea Hubert, Peter Huppke, Ralf A Husain, Sabine Illsinger, Eva Jansen, Lisa Jung, Nadja Kaiser, Stefan Kappel, Katharina Bolte, Johannes Koch, Stefan Kölker, Brigitte Korschinsky, Franziska Kostede, Karsten Krause, Annina Lang, Irene Lange, Thorsten Langer, Yvonne Lechner, Helmar Lehmann, Christine Leypold, Paul Lingor, Jaqueline Lipka, Wolfgang Löscher, Antje Luiking, Gerrit Machetanz, Eva Malm, Kyriakos Martakis, Bettina Menzen, Moritz Metelmann, Gerd Meyer Zu Hörste, Federica Montagnese, Kathrin Mörtlbauer, Petra Müller, Anne Müller, Anja Müller, Lars Müschen, Moritz Niesert, Josefine Pauschek, Elke Pernegger, Veronika Pilshofer, Barbara Plecko, Jürgen Pollok, Martin Preisel, Manuel Pühringer, Anna Lisa Quinten, Sabine Raffler, Barbara Ramadan, Mika Rappold, Christian Rauscher, Kerstin Reckmann, Christof Reihle, Tabea Reinhardt, Melanie Röder, Doris Roland-Schäfer, Erdmute Roth, Lena Ruß, Afshin Saffari, Mareike Schimmel, Kurt Schlachter, Melina Schlag, Beate Schlotter-Weigel, Joanna Schneider, Jan-Christoph Schöne-Bake, David Schorling, Isabella Schreiner, Stephanie Schüssler, Michaela Schwarzbach, Anette Schwerin-Nagel, Michaela Schwippert, Luisa Semmler, Karin Smuda, Alina Sprenger-Svacina, Theresa Stadler, Paula Steffens, Daniela Steuernagel, Benjamin Stolte, Andreas Thimm, Elke Tiefenthaler, Raffi Topakian, Lieske van der Stam, Katia Vettori, Peter Vollmann, Matthias Vorgerd, Claudia Weiß, Deike Weiss, Stephan Wenninger, Svea Werring, Maria Wessel, Ute Weyen, Sabine Wider, Nils Ole Wiebe, Gert Wiegand, Anna Wiesenhofer, Sarah Wiethoff, Corinna Wirner, Camilla Wohnrade, Gilbert Wunderlich, Michael Zemlin, Joachim Zobel

Affiliations

¹ Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
² Institute of Medical Biometry and Statistics, Faculty of Medicine and Medical Center - University of Freiburg, Freiburg, Germany.
³ Clinical Trials Unit, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
⁴ Clinic Favoriten, Department of Pediatrics, Vienna, Austria.
⁵ Department of Neurology, and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, Essen, Germany.
⁶ Department of Neuropediatrics and Neuromuscular Centre for children and Adolescents, Center for Translational Neuro- and Behavioral Sciences, University of Duisburg-Essen, Essen, Germany.
⁷ Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.
⁸ Department of Neurology, Christian-Albrechts-University of Kiel, Kiel, Germany.
⁹ Department of Pediatric Neurology and Developmental Medicine and LMU Center for Children with Medical Complexity, Dr. von Hauner Children's Hospital, LMU Hospital, Ludwig-Maximilians-University, Munich, Germany.
¹⁰ Department of Pediatrics I, Division of Pediatric Neurology, Medical University of Innsbruck, Innsbruck, Austria.
¹¹ Ordensklinikum Linz, Barmherzige Schwestern, Department of Pediatrics and Adulescent Medicine, Linz, Austria.
¹² Department of Neurology, School of Medicine, Technical University of Munich, Munich, Germany.
¹³ Department of Pediatric Neurology, Saarland University Hospital, Homburg, Germany.
¹⁴ Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
¹⁵ Department of Neurology, University Hospital Carl Gustav Carus, Dresden, Germany.
¹⁶ Department of Child Neurology, Justus-Liebig University, Giessen, Germany.
¹⁷ Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
¹⁸ Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
¹⁹ Department of Neurology, University of Rostock, Rostock, Germany.
²⁰ Department of Neurology, University Medical Center Göttingen, Göttingen, Germany.
²¹ Ruhr-Universität Bochum, St. Josef-Hospital, Abteilung für Neuropädiatrie und Sozialpädiatrie, Universitätsklinik für Kinder- und Jugendmedizin, Bochum, Germany.
²² Children's Hospital Amsterdamer Straße, Kliniken der Stadt Köln, Cologne, Germany.
²³ Department of Pediatrics und Neuropediatrics, DRK Kliniken Berlin, Berlin, Germany.
²⁴ Department of Neurology, University Hospital Halle, Martin-Luther-University Halle-Wittenberg, Halle, Germany.
²⁵ Neuromuscular Diseases Unit / ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland.
²⁶ Department of Neurology, Hannover Medical School, Hannover, Germany.
²⁷ Department of Neurology, University Hospital Jena, Jena, Germany.
²⁸ Pediatrics and Adolescent Medicine, Pediatric Neurology, University Medical Center Augsburg, Augsburg, Germany.
²⁹ Fachbereich Neurologie, DKD Helios Klinik Wiesbaden, Wiesbaden, Germany.
³⁰ Department of Pediatric Neurology, Klinikum Kassel, Kassel, Germany.
³¹ Abteilung Neuropaediatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
³² Department of Neurology, Klinikum Klagenfurt am Wörthersee, Klagenfurt am Wörthersee, Austria.
³³ Department of Pediatrics and Adolescent Medicine, Johannes Kepler University / Hospital, Linz, Austria.
³⁴ Department of Pediatrics, Landesklinikum Baden-Mödling, Standort Mödling, Germany.
³⁵ Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany.
³⁶ Department of Neurology, Friedrich-Alexander University Erlangen-Nuremberg (FAU), Germany.
³⁷ Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
³⁸ Department of Pediatric Neurology and Center for Chronically Sick Children, Charité - University Medicine Berlin, Berlin, Germany.
³⁹ Department of Paediatrics and Pediatric Neurology, University Medical Centre, Georg August University Göttingen, Göttingen, Germany.
⁴⁰ Department of Neurology, University Hospital Würzburg, Würzburg, Germany.
⁴¹ Department of Neuropediatrics and Metabolic Medicine, University Hospital Heidelberg, Heidelberg, Germany.
⁴² Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada.
⁴³ Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, ON, Canada.
⁴⁴ Brain and Mind Research Institute, University of Ottawa, Ottawa, ON, Canada.

PMID: 36565133
PMCID: PMC9881023
DOI: 10.3233/JND-221600

Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study

Astrid Pechmann et al. J Neuromuscul Dis. 2023.

. 2023;10(1):29-40.

doi: 10.3233/JND-221600.

Authors

Collaborators

SMArtCARE Study Group:
Lisa Ameshofer, Barbara Andres, Daniela Angelova-Toshkina, Daniela Banholzer, Christina Bant, Petra Baum, Sandra Baumann, Ute Baur, Bettina Behring, Julia Bellut, Astrid Bertsche, Andrea Bevot, Jasmin Bischofberger, Lisa Bitzan, Bogdan Bjelica, Markus Blankenburg, Sandra Böger, Friederike Bonetti, Anke Bongartz, Svenja Brakemeier, Lisa Bratka, Nathalie Braun, Sarah Braun, Brigitte Brauner, Christa Bretschneider, Nadine Burgenmeister, Bea Burke, Sebahattin Cirak, Isabell Cordts, Andrea Dall, Heike de Vries, Adela Della Marina, Jonas Denecke, Marcus Deschauer, Zylfie Dibrani, Uta Diebold, Lutz Dondit, Jessica Drebes, Joenna Driemeyer, Vladimir Dukic, Matthias Eckenweiler, Astrid Eisenkölbl, Mirjam Eminger, Michal Fischer, Cornelia Fischer, Maren Freigang, Philippa Gaiser, Andrea Gangfuß, Gehrke Tasch, Stephanie Geitmann, Annette George, Magdalena Gosk-Tomek, Susanne Grinzinger, Kristina Gröning, Martin Groß, Anne-Katrin Güttsches, Anna Hagenmeyer, Hans Hartmann, Julia Haverkamp, Miriam Hiebeler, Annegret Hoevel, Georg Friedrich Hoffmann, Britta Holtkamp, Dorothea Holzwarth, Annette Homma, Veronka Horber, Viola Horneff, Carolin Hörnig, Anna Hotter, Andrea Hubert, Peter Huppke, Ralf A Husain, Sabine Illsinger, Eva Jansen, Lisa Jung, Nadja Kaiser, Stefan Kappel, Katharina Bolte, Johannes Koch, Stefan Kölker, Brigitte Korschinsky, Franziska Kostede, Karsten Krause, Annina Lang, Irene Lange, Thorsten Langer, Yvonne Lechner, Helmar Lehmann, Christine Leypold, Paul Lingor, Jaqueline Lipka, Wolfgang Löscher, Antje Luiking, Gerrit Machetanz, Eva Malm, Kyriakos Martakis, Bettina Menzen, Moritz Metelmann, Gerd Meyer Zu Hörste, Federica Montagnese, Kathrin Mörtlbauer, Petra Müller, Anne Müller, Anja Müller, Lars Müschen, Moritz Niesert, Josefine Pauschek, Elke Pernegger, Veronika Pilshofer, Barbara Plecko, Jürgen Pollok, Martin Preisel, Manuel Pühringer, Anna Lisa Quinten, Sabine Raffler, Barbara Ramadan, Mika Rappold, Christian Rauscher, Kerstin Reckmann, Christof Reihle, Tabea Reinhardt, Melanie Röder, Doris Roland-Schäfer, Erdmute Roth, Lena Ruß, Afshin Saffari, Mareike Schimmel, Kurt Schlachter, Melina Schlag, Beate Schlotter-Weigel, Joanna Schneider, Jan-Christoph Schöne-Bake, David Schorling, Isabella Schreiner, Stephanie Schüssler, Michaela Schwarzbach, Anette Schwerin-Nagel, Michaela Schwippert, Luisa Semmler, Karin Smuda, Alina Sprenger-Svacina, Theresa Stadler, Paula Steffens, Daniela Steuernagel, Benjamin Stolte, Andreas Thimm, Elke Tiefenthaler, Raffi Topakian, Lieske van der Stam, Katia Vettori, Peter Vollmann, Matthias Vorgerd, Claudia Weiß, Deike Weiss, Stephan Wenninger, Svea Werring, Maria Wessel, Ute Weyen, Sabine Wider, Nils Ole Wiebe, Gert Wiegand, Anna Wiesenhofer, Sarah Wiethoff, Corinna Wirner, Camilla Wohnrade, Gilbert Wunderlich, Michael Zemlin, Joachim Zobel

Affiliations

¹ Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
² Institute of Medical Biometry and Statistics, Faculty of Medicine and Medical Center - University of Freiburg, Freiburg, Germany.
³ Clinical Trials Unit, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
⁴ Clinic Favoriten, Department of Pediatrics, Vienna, Austria.
⁵ Department of Neurology, and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, Essen, Germany.
⁶ Department of Neuropediatrics and Neuromuscular Centre for children and Adolescents, Center for Translational Neuro- and Behavioral Sciences, University of Duisburg-Essen, Essen, Germany.
⁷ Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.
⁸ Department of Neurology, Christian-Albrechts-University of Kiel, Kiel, Germany.
⁹ Department of Pediatric Neurology and Developmental Medicine and LMU Center for Children with Medical Complexity, Dr. von Hauner Children's Hospital, LMU Hospital, Ludwig-Maximilians-University, Munich, Germany.
¹⁰ Department of Pediatrics I, Division of Pediatric Neurology, Medical University of Innsbruck, Innsbruck, Austria.
¹¹ Ordensklinikum Linz, Barmherzige Schwestern, Department of Pediatrics and Adulescent Medicine, Linz, Austria.
¹² Department of Neurology, School of Medicine, Technical University of Munich, Munich, Germany.
¹³ Department of Pediatric Neurology, Saarland University Hospital, Homburg, Germany.
¹⁴ Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
¹⁵ Department of Neurology, University Hospital Carl Gustav Carus, Dresden, Germany.
¹⁶ Department of Child Neurology, Justus-Liebig University, Giessen, Germany.
¹⁷ Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
¹⁸ Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
¹⁹ Department of Neurology, University of Rostock, Rostock, Germany.
²⁰ Department of Neurology, University Medical Center Göttingen, Göttingen, Germany.
²¹ Ruhr-Universität Bochum, St. Josef-Hospital, Abteilung für Neuropädiatrie und Sozialpädiatrie, Universitätsklinik für Kinder- und Jugendmedizin, Bochum, Germany.
²² Children's Hospital Amsterdamer Straße, Kliniken der Stadt Köln, Cologne, Germany.
²³ Department of Pediatrics und Neuropediatrics, DRK Kliniken Berlin, Berlin, Germany.
²⁴ Department of Neurology, University Hospital Halle, Martin-Luther-University Halle-Wittenberg, Halle, Germany.
²⁵ Neuromuscular Diseases Unit / ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland.
²⁶ Department of Neurology, Hannover Medical School, Hannover, Germany.
²⁷ Department of Neurology, University Hospital Jena, Jena, Germany.
²⁸ Pediatrics and Adolescent Medicine, Pediatric Neurology, University Medical Center Augsburg, Augsburg, Germany.
²⁹ Fachbereich Neurologie, DKD Helios Klinik Wiesbaden, Wiesbaden, Germany.
³⁰ Department of Pediatric Neurology, Klinikum Kassel, Kassel, Germany.
³¹ Abteilung Neuropaediatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
³² Department of Neurology, Klinikum Klagenfurt am Wörthersee, Klagenfurt am Wörthersee, Austria.
³³ Department of Pediatrics and Adolescent Medicine, Johannes Kepler University / Hospital, Linz, Austria.
³⁴ Department of Pediatrics, Landesklinikum Baden-Mödling, Standort Mödling, Germany.
³⁵ Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany.
³⁶ Department of Neurology, Friedrich-Alexander University Erlangen-Nuremberg (FAU), Germany.
³⁷ Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
³⁸ Department of Pediatric Neurology and Center for Chronically Sick Children, Charité - University Medicine Berlin, Berlin, Germany.
³⁹ Department of Paediatrics and Pediatric Neurology, University Medical Centre, Georg August University Göttingen, Göttingen, Germany.
⁴⁰ Department of Neurology, University Hospital Würzburg, Würzburg, Germany.
⁴¹ Department of Neuropediatrics and Metabolic Medicine, University Hospital Heidelberg, Heidelberg, Germany.
⁴² Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada.
⁴³ Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, ON, Canada.
⁴⁴ Brain and Mind Research Institute, University of Ottawa, Ottawa, ON, Canada.

PMID: 36565133
PMCID: PMC9881023
DOI: 10.3233/JND-221600

Abstract

Background and objectives: Disease progression in patients with spinal muscular atrophy (SMA) has changed dramatically within the past years due to the approval of three different disease-modifying treatments. Nusinersen was the first drug to be approved for the treatment of SMA patients. Clinical trials provided data from infants with SMA type 1 and children with SMA type 2, but there is still insufficient evidence and only scarcely reported long-term experience for nusinersen treatment in ambulant patients. Here, we report data from the SMArtCARE registry of ambulant patients under nusinersen treatment with a follow-up period of up to 38 months.

Methods: SMArtCARE is a disease-specific registry in Germany, Austria and Switzerland. Data are collected as real-world data during routine patient visits. Our analysis included all patients under treatment with nusinersen able to walk independently before start of treatment with focus on changes in motor function.

Results: Data from 231 ambulant patients were included in the analysis. During the observation period, 31 pediatric walkers (27.2%) and 31 adult walkers (26.5%) experienced a clinically meaningful improvement of≥30 m in the 6-Minute-Walk-Test. In contrast, only five adult walkers (7.7%) showed a decline in walking distance≥30 m, and two pediatric walkers (1.8%) lost the ability to walk unassisted under treatment with nusinersen. HFMSE and RULM scores improved in pediatric and remained stable in adult patients.

Conclusion: Our data demonstrate a positive effect of nusinersen treatment in most ambulant pediatric and adult SMA patients. We not only observed a stabilization of disease progression or lack of deterioration, but clinically meaningful improvements in walking distance.

Keywords: SMArtCARE; Spinal muscular atrophy; ambulant; nusinersen; walker.

PubMed Disclaimer

Conflict of interest statement

A. Pechmann received compensation for advisory boards, training activities and research grants from Novartis and Biogen. M. Schimmel received compensation for advisory boards from Roche; A. Blaschek received personal fees for advisory board services / speakers honoraria from Novartis, Roche, Ad medicum, Sanofi Genzyme and Avexis; JC Koch received compensation for advisory boards and talks from Biogen and Roche; D. Zeller received compensation for scientific consultancy from Novartis, for advisory boards from Biogen, and for training activities from Desitin; M. Baumann received compensation for advisory boards and speakers honoraria from Novartis, Biogen and Roche; R. Trollmann reports personal fees from Desitin Pharma, Novartis, PTC Therapeutics; M. Weiler received compensation for advisory boards, consultant and speaker honoraria and/or financial support for conference attendances from Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, Pfizer, Roche, and Sobi; C. Neuwirth received compensation for advisory boards and training activities by Biogen, Roche, Genzyme and Mitsubishi Tanabe; J. Friese received compensation for an advisory board from Novartis Gene Therapies; B. Schrank received compensation for advisory board from Biogen; M. Flotats-Bastardas received compensation for advisory boards from Biogen, Novartis and Roche; M.C. Walter received compensation for advisory boards, funding for travel or speaker honoraria, and research support from Biogen, Novartis, and Roche; R. Günther received compensation for advisory boards and research support from Biogen and compensation for advisory boards from Hofmann La-Roche; M. Türk received compensation for a presentation and an advisory board from Sanofi Genzyme; T. Hagenacker received compensation for advisory board, lectures and research grants from Biogen, Roche and Novartis; J. Johansen received honoraria for advisory board participation and/or lectures and/or manuscript writing from Avexis/Novartis, Biogen, Roche, PTC, Pfizer and Sarepta Th., respectively, and financial support from Biogen for the SMArtCARE registry (paid to institution); S. Petri received compensation for advisory boards from Biogen, Roche and Zambon, speaker honoraria from Biogen, Roche, Italfarmaco and Novartis, grants from the German Israeli Foundation (GIF), German Neuromuscular Society (DGM) and Neurodegenerative Research (NDR); C. Stadler received fees for speeches & participation in discussions from Merz Pharma; B. Becker received financial support for a publication from Biogen; C. Kamm received honoraria as a speaker and advisory board member from Biogen, Roche and Ipsen; C. Köhler received honoraria from Roche, PTC, Biogen and Genzyme; M. Smitka received funding for educational activities and participation in advisory boards concering SMA from Novartis Gene Therapies, Biogen and Roche; H. Kölbel received compensation for presentations and consultancy from Avexis, Biogen, Sanofi, Pfizer, Novartis, and Roche; A. Ziegler receives honoraria for advisory boards and speaker honoraria from Novartis Gene Therapies, Roche Pharma and Biogen. His institution receives research grants from Biogen; G. Bernert receives research suppert from PTC and honoraria from Novartis Gene Therapies, PTC, Biogen, Roche, Pfizer, Santhera; E. Wilichowski received compensation for advisory boards from Biogen, Novartis and Roche; H. Lochmüller received consultancy and financial support for research projects and clinical trials from Amplo Biotechnology, AMO Pharma, argenx, Biogen, Desitin, Fulcrum Therapeutics, Harmony Biosciences, KYE Pharmaceuticals, Milo Biotechnology, Novartis, Pfizer, PTC Therapeutics, Hoffman-La Roche Limited, Sanofi-Genzyme, Santhera, Sarepta, Satellos, Spark Therapeutics and Ultragenyx. HL is the Editor-in-chief for the Journal of Neuromuscular Diseases (IOS Press); J. Kirschner received support from Biogen, Novartis, Roche and ScholarRock for consultancy, educational activities and/or clinical research.

Figures

**Fig. 1**
Longitudinal progression of the 6MWT. 6MWT for pediatric (blue) and adult (red) walkers. Data are listed as mean and 99% confidence interval. Available patients at baseline, m14, m26 and m38 are added. For a group size fewer than 10 patients no data are shown. As the performance of the 6MWT is not mandatory within the SMArtCARE data collection, data are not available for all patients at all time-points.

**Fig. 2**
Responder analysis 6MWT. Responder analysis for pediatric walkers and adult walkers. Colors indicate response groups according to changes in 6MWT per time-period (baseline-m14, m14-m26, m26-m38). Lines between columns indicate the progression of patients over time with improvements or worsening in walking distance.

**Fig. 3**
Longitudinal progression of HFMSE and RULM score. A HMFSE and B RULM score for pediatric (blue) and adult walkers (red). Data are listed as mean and 99% confidence interval. Available patients at baseline, m14, m26 and m38 are added. For a group size fewer than 10 patients no data are depicted.

**Fig. 4**
Ceiling effect of outcome measures. Longitudinal progression for HFMSE, RULM and 6WMT in pediatric (A) and adult walkers (B). Lines represent individual patients. Red horizontal lines reflect a ceiling effect with≥60 points in HFMSE,≥35 points in RULM and≥500 m in 6MWT.

See this image and copyright information in PMC

References

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Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study

Collaborators

Affiliations

Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study

Authors

Collaborators

Affiliations

Abstract

Conflict of interest statement

Figures

References

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