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Open Access
Peer-reviewed
Research Article
Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study
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Aurélie Chabanon,
Roles Data curation, Methodology, Project administration, Visualization, Writing – original draft, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Andreea Mihaela Seferian,
Roles Investigation, Resources, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Aurore Daron,
Roles Investigation, Resources, Writing – review & editing
Affiliation Centre de Référence des Maladies Neuromusculaires, CHU de Liège, Belgium
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Yann Péréon,
Roles Investigation, Resources, Writing – review & editing
Affiliation Centre de Référence Maladies Neuromusculaires Atlantique-Occitanie-Caraïbes, Hôpital Hôtel-Dieu, Nantes, France
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Claude Cances,
Roles Investigation, Resources, Writing – review & editing
Affiliations Centre de Référence des Maladies Neuromusculaires, Hôpital des Enfants, Toulouse, France, Unité de neurologie pédiatrique, Hôpital des Enfants, Toulouse, France
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Carole Vuillerot,
Roles Investigation, Resources, Writing – review & editing
Affiliation Service de rééducation pédiatrique infantile”L’Escale”, Hôpital Mère Enfant, CHU-Lyon, Lyon, France
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Liesbeth De Waele,
Roles Investigation, Resources, Writing – review & editing
Affiliations Department of Pediatric Neurology, University Hospitals Leuven, Leuven, Belgium, Department of Development and Regeneration, KU Leuven Kulak Kortijk, Kortrijk, Belgium
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Jean-Marie Cuisset,
Roles Investigation, Resources, Writing – review & editing
Affiliations Centre de Référence des Maladies Neuromusculaires, Hôpital Roger Salengro, Lille, France, Service de Neuropédiatrie, Hôpital Roger Salengro, Lille, France
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Vincent Laugel,
Roles Investigation, Resources, Writing – review & editing
Affiliation Neuropédiatrie/INSERM CIC 1434, CHU Strasbourg Hautepierre, Strasbourg, France
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Ulrike Schara,
Roles Investigation, Resources, Writing – review & editing
Affiliation Paediatric neurology and neuromuscular center, University of Essen, Essen, Germany
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Teresa Gidaro,
Roles Investigation, Resources, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Stéphanie Gilabert,
Roles Investigation, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Jean-Yves Hogrel,
Roles Conceptualization, Data curation, Methodology, Resources, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Pierre-Yves Baudin,
Roles Resources
Affiliation Consultants for Research in Imaging and Spectroscopy (CRIS), Tournai, Belgium
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Pierre Carlier,
Roles Investigation, Methodology, Resources, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Emmanuel Fournier,
Roles Data curation, Investigation, Methodology, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Linda Pax Lowes,
Roles Data curation, Methodology, Resources, Writing – review & editing
Affiliation Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA
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Nicole Hellbach,
Roles Methodology, Resources, Writing – review & editing
Affiliation Roche Pharmaceutical Research and Early Development, Roche Innovation Center, Basel, Switzerland
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Timothy Seabrook,
Roles Conceptualization, Funding acquisition, Methodology, Supervision, Writing – review & editing
Affiliation Roche Pharmaceutical Research and Early Development, Roche Innovation Center, Basel, Switzerland
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Elie Toledano,
Roles Conceptualization, Funding acquisition, Methodology, Supervision, Writing – review & editing
Affiliation Institut Roche, Paris, France
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Mélanie Annoussamy,
Roles Conceptualization, Formal analysis, Funding acquisition, Methodology, Project administration, Supervision, Writing – review & editing
Affiliation Institute of Myology, GH Pitié Salpêtrière, Paris, France
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Laurent Servais ,
Roles Conceptualization, Funding acquisition, Investigation, Methodology, Resources, Supervision, Writing – review & editing
* E-mail: l.servais@institut-myologie.org
Affiliations Institute of Myology, GH Pitié Salpêtrière, Paris, France, Centre de Référence des Maladies Neuromusculaires, CHU de Liège, Belgium, Service de Pédiatrie, CHU de Liège, Liège, Belgium
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the NatHis-SMA study group
¶Membership of the NatHis-SMA study group is provided in the Acknowledgments.
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Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study
- Aurélie Chabanon,
- Andreea Mihaela Seferian,
- Aurore Daron,
- Yann Péréon,
- Claude Cances,
- Carole Vuillerot,
- Liesbeth De Waele,
- Jean-Marie Cuisset,
- Vincent Laugel,
- Ulrike Schara
- Published: July 26, 2018
- https://doi.org/10.1371/journal.pone.0201004