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Authors
Pechmann, Astrid https://orcid.org/0000-0001-9579-2343
Behrens, Max
Dörnbrack, Katharina
Tassoni, Adrian
Wenzel, Franziska
Stein, Sabine
Vogt, Sibylle
Zöller, Daniela
Bernert, Günther
Hagenacker, Tim
Schara-Schmidt, Ulrike
Walter, Maggie C.
Bertsche, Astrid
Vill, Katharina
Baumann, Matthias
Baumgartner, Manuela
Cordts, Isabell
Eisenkölbl, Astrid
Flotats-Bastardas, Marina
Friese, Johannes
Günther, René
Hahn, Andreas
Horber, Veronka
Husain, Ralf A.
Illsinger, Sabine
Jahnel, Jörg
Johannsen, Jessika
Köhler, Cornelia
Kölbel, Heike
Müller, Monika
von Moers, Arpad
Schwerin-Nagel, Annette
Reihle, Christof
Schlachter, Kurt
Schreiber, Gudrun
Schwartz, Oliver
Smitka, Martin
Steiner, Elisabeth
Trollmann, Regina
Weiler, Markus
Weiß, Claudia
Wiegand, Gert
Wilichowski, Ekkehard
Ziegler, Andreas
Lochmüller, Hanns
Kirschner, Janbernd
,
Ameshofer, Lisa
Andres, Barbara
Angelova-Toshkina, Daniela
Banholzer, Daniela
Bant, Christina
Baum, Petra
Baumann, Sandra
Baur, Ute
Becker, Benedikt
Behring, Bettina
Bellut, Julia
Bevot, Andrea
Bischofberger, Jasmin
Bitzan, Lisa
Bjelica, Bogdan
Blankenburg, Markus
Böger, Sandra
Bonetti, Friederike
Bongartz, Anke
Brakemeier, Svenja
Bratka, Lisa
Braun, Nathalie
Braun, Sarah
Brauner, Brigitte
Bretschneider, Christa
Burgenmeister, Nadine
Burke, Bea
Cirak, Sebahattin
Dall, Andrea
de Vries, Heike
Marina, Adela Della
Denecke, Jonas
Deschauer, Marcus
Dibrani, Zylfie
Diebold, Uta
Dondit, Lutz
Drebes, Jessica
Driemeyer, Joenna
Dukic, Vladimir
Eckenweiler, Matthias
Eminger, Mirjam
Fischer, Michal
Fischer, Cornelia
Freigang, Maren
Gaiser, Philippa
Gangfuß, Andrea
Geitmann, Stephanie
George, Annette
Gosk-Tomek, Magdalena
Grinzinger, Susanne
Gröning, Kristina
Groß, Martin
Güttsches, Anne-Katrin
Hagenmeyer, Anna
Hartmann, Hans
Haverkamp, Julia
Hiebeler, Miriam
Hoevel, Annegret
Hoffmann, Georg Friedrich
Holtkamp, Britta
Holzwarth, Dorothea
Homma, Annette
Horneff, Viola
Hörnig, Carolin
Hotter, Anna
Hubert, Andrea
Huppke, Peter
Jansen, Eva
Jung, Lisa
Kaiser, Nadja
Kappel, Stefan
Katharina, Bolte
Koch, Johannes
Kölke, Stefan
Korschinsky, Brigitte
Kostede, Franziska
Krause, Karsten
Küpper, Hanna
Lang, Annina
Lange, Irene
Langer, Thorsten
Lechner, Yvonne
Lehmann, Helmar
Leypold, Christine
Lingor, Paul
Lipka, Jaqueline
Löscher, Wolfgang
Luiking, Antje
Machetanz, Gerrit
Malm, Eva
Martakis, Kyriakos
Menzen, Bettina
Metelmann, Moritz
Meyer zu Hörste, Gerd
Montagnese, Federica
Mörtlbauer, Kathrin
Müller, Petra
Müller, Anne
Müller, Anja
Müschen, Lars
Neuwirth, Christoph
Niesert, Moritz
Pauschek, Josefine
Pernegger, Elke
Petri, Susanne
Pilshofer, Veronika
Plecko, Barbara
Pollok, Jürgen
Preisel, Martin
Pühringer, Manuel
Quinten, Anna Lisa
Raffler, Sabine
Ramadan, Barbara
Rappold, Mika
Rauscher, Christian
Reckmann, Kerstin
Reinhardt, Tabea
Röder, Melanie
Roland-Schäfer, Doris
Roth, Erdmute
Ruß, Lena
Saffari, Afshin
Schimmel, Mareike
Schlag, Melina
Schlotter-Weigel, Beate
Schneider, Joanna
Schöne-Bake, Jan-Christoph
Schorling, David
Schreiner, Isabella
Schüssler, Stephanie
Schwarzbach, Michaela
Schwippert, Michaela
Semmler, Luisa
Smuda, Karin
Sprenger-Svacina, Alina
Stadler, Theresa
Steffens, Paula
Steuernagel, Daniela
Stolte, Benjamin
Stoltenburg, Corinna
Tasch, Gehrke
Thimm, Andreas
Tiefenthaler, Elke
Topakian, Raffi
Türk, Matthias
van der Stam, Lieske
Vettori, Katia
Vollmann, Peter
Vorgerd, Matthias
Weiss, Deike
Wenninger, Stephan
Werring, Svea
Wessel, Maria
Weyen, Ute
Wider, Sabine
Wiebe, Nils Ole
Wiesenhofer, Anna
Wiethoff, Sarah
Wirner, Corinna
Wohnrade, Camilla
Wunderlich, Gilbert
Zeller, Daniel
Zemlin, Michael
Zobel, Joachim
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Funding
Funding for this research was provided by:
Biogen
Novartis Gene Therapies
Universitätsklinikum Freiburg
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Article History
Received: 23 June 2022
Accepted: 4 October 2022
First Online: 23 October 2022
Declarations
:
: Ethics approval of the central ethics committee has been obtained [Ethics Committee of the University of Freiburg, Germany (EK 56/18)], and local ethics approvals were obtained from all participating centers. All patients or caregivers signed an informed consent form prior to being enrolled in the registry.
: Not applicable.
: A. Pechmann received compensation for advisory boards, training activities and research grants from Novartis and Biogen. C. Weiß received compensation for advisory boards, publications and/or presentations from Novartis, Biogen, and Roche. M. Baumann received compensation for advisory boards and speakers honoraria from Novartis, Biogen and Roche. M. Flotats-Bastardas received compensation for advisory boards from Biogen, Novartis 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. S. Illsinger received compensation for advisory board/consultant work from Novartis. A. Bertsche reports grants from UCB Pharma GmbH and honoraria for speaking engagements and advisory boards from Biogen GmbH, Desitin Arzneimittel GmbH, Eisai GmbH, GW Pharma GmbH, Neuraxpharm GmbH, Shire/Takeda GmbH, UCB Pharma GmbH, and ViroPharma GmbH. A. Eisenkölbl received honoraria for advisory boards, interviews and speaking engegements from Biogen, Novartis and Roche. K. Vill received speaker honoraria and travel expenses from Novartis, Biogen and Santhera. M.C. Walter received compensation for advisory boards, funding for travel or speaker honoraria, and research support from Biogen, Novartis, and Roche. J. Johannsen received honoraria for advisory board participation and/or lectures and/or manuscript writing from Avexis/Novartis, Biogen, Roche, PTC, Pfizer and Sarepta Th., respectively. C. Reihle reports travel expenses from Biogen. R. Günther received compensation for advisory boards and research support from Biogen and compensation for advisory boards from Hofmann La-Roche. T. Hagenacker received compensation for advisory boards, lectures and research grants from Novartis, Biogen and Roche. V. Horber participated in workshops and received compensation for advisory boards from Novartis and Biogen. C. Köhler received honoraria from Roche, PTC, Biogen and Genzyme. M. Müller received compensation for expert meetings and training vom Norvartis, Biogen and Roche. O. Schwarz received compensation for advisory boards and training activities and research grants from Novartis and Biogen. I. Cordts received personal fees from Biogen (speaker’s honoraria, participation in advisory boards) outside the submitted work. M. Smitka received funding for educational activities and participation in advisory boards concerning SMA from Novartis Gene Therapies, Biogen and Roche. A. Schwerin-Nagel received compensation for an advisory board from Novartis Gene Therapies. H. Kölbel received compensation for presentations and consultancy from Avexis, Biogen, Sanofi, Pfizer, Novartis, and Roche. K. Schlachter received compensation for advisory boards from Novartis, Biogen and Roche. J. Friese received compensation for an advisory board from Novartis Gene Therapies. 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.
