换一批Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence.
第一作者：
Claudia,Weiß
第一单位：
Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, Germany.
作者：
Claudia,Weiß [1]
;
Katharina,Vill [2]
;
Matthias,Baumann [3]
;
Günther,Bernert [4]
;
Astrid,Blaschek [2]
;
Astrid,Eisenkölbl [5]
;
Marina,Flotats-Bastardas [6]
;
Johannes,Friese [7]
;
Claudia,Ganter [8]
;
Klaus,Goldhahn [9]
;
Andreas,Hahn [10]
;
Maja,von der Hagen [11]
;
Hans,Hartmann [12]
;
Oswald,Hasselmann [13]
;
Veronka,Horber [14]
;
Ralf A,Husain [15]
;
Sabine,Illsinger [12]
;
David,Jacquier [16]
;
Jessika,Johannsen [17]
;
Cornelia,Köhler [18]
;
Heike,Kölbel [19]
;
Michael,Kolodzig [20]
;
Andrea,Klein [21]
;
Astrid,Pechmann [22]
;
Arpad,von Moers [9]
;
Wolfgang,Müller-Felber [2]
;
Christian,Rauscher [23]
;
Ulrike,Schara-Schmidt [19]
;
Gudrun,Schreiber [24]
;
Oliver,Schwartz [25]
;
Joachim,Sproß [26]
;
Georg M,Stettner [27]
;
Corinna,Stoltenburg [1]
;
Eva,Stumpe [28]
;
Regina,Trollmann [29]
;
Gert,Wiegand [30]
;
Ekkehard,Wilichowski [31]
;
Janbernd,Kirschner [22]
;
Andreas,Ziegler [32]
;
collaborators and members of the INTEGRATE ATMP consortium [33]
作者单位：
Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Augustenburger Platz 1, 13353 Berlin, Germany.
[1]
Department of Paediatric Neurology and Developmental Medicine, Ludwig Maximilian University of Munich (LMU), Hauner Children's Hospital, Munich, Germany.
[2]
Division of Pediatric Neurology, Department of Pediatrics I, Medical University of Innsbruck, Innsbruck, Austria.
[3]
Department of Pediatrics, Klinik Favoriten, Vienna, Austria.
[4]
Department of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Kepler University Hospital, 4020 Linz, Austria.
[5]
Department of General Pediatrics and Neonatology, Division of Neuropediatrics, Saarland University, Homburg Saar, Germany.
[6]
Department of Child Neurology, University Hospital Bonn, Bonn, Germany.
[7]
Medical Faculty Heidelberg, INTEGRATE ATMP Project, Heidelberg University, Heidelberg, Germany.
[8]
Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Berlin, Germany.
[9]
Department of Child Neurology, University Hospital Gießen, Gießen, Germany.
[10]
Department of Neuropediatrics, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
[11]
Clinic for Pediatric Kidney-, Liver-, Metabolic and Neurological Diseases, Hannover Medical School, Hannover, Germany.
[12]
Department of Pediatric Neurology, Children's hospital Ostschweiz, St. Gallen, Switzerland.
[13]
Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
[14]
Department of Neuropediatrics, Jena University Hospital, Jena, Germany.
[15]
Pediatric Neurology and Neurorehabilitation unit, Woman-Mother-Child Department, Lausanne University Hospital, Lausanne, Switzerland.
[16]
Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
[17]
Children's Hospital St Josef, University Hospital Ruhr-University Bochum, Bochum, Germany.
[18]
Department of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro and Behavioral Sciences, University Duisburg-Essen, Essen, Germany.
[19]
Deutsche Muskelstiftung, Karlsruhe, Germany.
[20]
Division of Neuropaediatrics, Development and Rehabilitation, Department of Paediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.
[21]
Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
[22]
Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria.
[23]
Department of Pediatric Neurology, Klinikum Kassel, Kassel, Germany.
[24]
Department of Pediatric Neurology, Muenster University Hospital, Münster, Germany.
[25]
German Society for Neuromuscular Diseases e.V. (DGM), Freiburg, Germany.
[26]
Neuromuscular Center Zürich and Department of Pediatric Neurology, University Children's Hospital, University of Zurich, Zürich, Switzerland.
[27]
SMA Europe e. V., Freiburg, Germany.
[28]
Department of Pediatrics, Division of Pediatric Neurology, Friedrich-Alexander University of Erlangen-Nürnberg, Erlangen, Germany.
[29]
Department of Pediatrics, Division of Neuropediatrics, Asklepios Klinik Hamburg Nord-Heidberg, Hamburg, Germany.
[30]
Department of Pediatrics and Adolescent Medicine, University Medical Center Göttingen, Göttingen, Germany.
[31]
Medical Faculty Heidelberg, Center for Pediatric and Adolescent Medicine, Department I, Division of Pediatric Neurology and Metabolic Medicine, Heidelberg University, Heidelberg, Germany.
[32]
INTEGRATE ATMP coordination team, Heidelberg, Germany.
[33]
DOI
10.1177/22143602251387019
PMID
41284552
发布时间
2025-11-24
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Journal of neuromuscular diseases
Journal of neuromuscular diseases
22143602251387019页
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