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Anzahl der Publikationen: 6
Zeitschriftenartikel
Weiß, Claudia; Ziegler, Andreas; Becker, Lena-Luise; Johannsen, Jessika; Brennenstuhl, Heiko; Schreiber, Gudrun; Flotats-Bastardas, Marina; Stoltenburg, Corinna; Hartmann, Hans; Illsinger, Sabine; Denecke, Jonas; Pechmann, Astrid; Müller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka, Martin; Stam, Lieske van der; Weiss, Katja; Winter, Benedikt; Goldhahn, Klaus; Plecko, Barbara; Horber, Veronka; Bernert, Günther; Husain, Ralf A.; Rauscher, Christian; Trollmann, Regina; Garbade, Sven F.; Hahn, Andreas; Hagen, Maja von der und Kaindl, Angela M.
(2022):
Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.
In: Lancet Child & Adolescent Health, Bd. 6, Nr. 1: S. 17-27
Ziegler, Andreas; Wilichowski, Ekkehard; Schara, Ulrike; Hahn, Andreas; Müller-Felber, Wolfgang; Johannsen, Jessika; Hagen, Maja von der; Moers, Arpad von; Stoltenburg, Corinna; Saffari, Afshin; Walter, Maggie C.; Husain, Ralf A.; Pechmann, Astrid; Köhler, Cornelia; Horber, Veronka; Schwartz, Oliver und Kirschner, Janbernd
(2020):
Handlungsempfehlungen zur Gentherapie der spinalen Muskelatrophie mit Onasemnogene Abeparvovec – AVXS-101.
In: Nervenarzt, Bd. 91, Nr. 6: S. 518-529
Janisch, Maria; Boehme, Kristin; Thiele, Simone; Bock, Annette; Kirschner, Janbernd; Schara, Ulrike; Walter, Maggie C.; Nolte-Buchholtz, Silke und Hagen, Maja von der
(2020):
Tasks and interfaces in primary and specialized palliative care for Duchenne muscular dystrophy - A patients' perspective.
In: Neuromuscular Disorders, Bd. 30, Nr. 12: S. 975-985
Villar-Quiles, Rocio N.; Hagen, Maja von der; Metay, Corinne; Gonzalez, Victoria; Donkervoort, Sandra; Bertini, Enrico; Castiglioni, Claudia; Chaigne, Denys; Colomer, Jaume; Cuadrado, Maria Luz; Visser, Marianne de; Desguerre, Isabelle; Eymard, Bruno; Goemans, Nathalie; Kaindl, Angela; Lagrue, Emmanuelle; Lutschg, Jurg; Malfatti, Edoardo; Mayer, Michele; Merlini, Luciano; Orlikowski, David; Reuner, Ulrike; Salih, Mustafa A.; Schlotter-Weigel, Beate; Stoetter, Mechthild; Straub, Volker; Topaloglu, Haluk; Urtizberea, J. Andoni; Kooi, Anneke van der; Wilichowski, Ekkehard; Romero, Norma B.; Fardeau, Michel; Bonnemann, Carsten G.; Estournet, Brigitte; Richard, Pascale; Quijano-Roy, Susana; Schara, Ulrike und Ferreiro, Ana
(2020):
The clinical, histologic, and genotypic spectrum ofSEPN1-related myopathy A case series.
In: Neurology, Bd. 95, Nr. 11, E1512-E1527
Dittrich, Sven; Graf, Erika; Trollmann, Regina; Neudorf, Ulrich; Schara, Ulrike; Heilmann, Antje; Hagen, Maja von der; Stiller, Brigitte; Kirschner, Janbernd; Dalla Pozza, Robert; Müller-Felber, Wolfgang; Weiss, Katja; Au, Katja von; Khalil, Markus; Motz, Reinald; Korenke, Christoph; Lange, Martina; Wilichowski, Ekkehard; Pattathu, Joseph; Ebinger, Friedrich; Wiechmann, Nicola; Schröder, Rolf; Halbfass, Julia; Webinger, Jasmin; Weise, Anja; Hermdobler, Franz; Nerad, Mateja; Shabaiek, Amira; Akin-Erdinc, Gueler; Greim, Verena; Böcker, Dorothee; Siepe, Stefanie; Schneider-Fuchs, Sabine; Egenhofer-Kummert, Brigitte; Burkhardt, Barbara; Neumann, Elena; Korinthenberg, Rudolf; Apitz, Christian; Freund, Matthias; Schumacher, Michael; Gravenhorst, Verena; Deppe, Daniela und Eichhorn, Joachim
(2019):
Effect and safety of treatment with ACE-inhibitor Enalapril and beta-blocker metoprolol on the onset of left ventricular dysfunction in Duchenne muscular dystrophy - a randomized, double-blind, placebo-controlled trial.
In: Orphanet Journal of Rare Diseases, Bd. 14, 105
Chamova, Teodora; Bichev, Stoyan; Todorov, Tihomir; Gospodinova, Mariana; Taneva, Ani; Kastreva, Kristina; Zlatareva, Dora; Krupev, Martin; Hadjiivanov, Rosen; Guergueltcheva, Velina; Grozdanova, Liliana; Tzoneva, Dochka; Hübner, Angela; Hagen, Maja von der; Schoser, Benedikt; Lochmüller, Hanns; Todorova, Albena und Tournev, Ivailo
(2018):
Limb girdle muscular dystrophy 2G in a religious minority of Bulgarian Muslims homozygous for the c.75G > A, p.Trp25X mutation.
In: Neuromuscular Disorders, Bd. 28, Nr. 8: S. 625-632