Search Results - Kaindl, Angela M
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Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized,... by Driedger, Jan Henje (Author) , Schröter, Julian (Author) , Hertzberg, Christoph (Author) , Weschke, Bernhard (Author) , Kaindl, Angela M. (Author) , Lücke, Thomas (Author) , Thiels, Charlotte (Author) , Klotz, Kerstin A. (Author) , Fazeli, Walid (Author) , Rostásy, Kevin (Author) , Wiethoff-Ubrig, Lucia (Author) , Kaiser, Olaf (Author) , Trollmann, Regina (Author) , Mammadova, Dilbar (Author) , Schubert-Bast, Susanne (Author) , Bach, Alexia (Author) , Eckenweiler, Matthias (Author) , Schönberger, Jan (Author) , Martakis, Kyriakos (Author) , Hahn, Andreas (Author) , Brockmann, Knut (Author) , Dreha-Kulaczewski, Steffi (Author) , Weiss, Deike (Author) , Denecke, Jonas (Author) , Muhle, Hiltrud (Author) , Arélin, Maria (Author) , Merkenschlager, Andreas (Author) , Borggräfe, Ingo (Author) , Roser, Timo (Author) , Ebrahimi-Fakhari, Daniel (Author) , Fiedler, Barbara (Author) , Schlump, Jan-Ulrich (Author) , Köster, Ilka (Author) , Korenke, Christoph (Author) , Alber, Michael (Author) , Ruf, Susanne (Author) , Feucht, Martha (Author) , Scholl, Theresa (Author) , Syrbe, Steffen (Author) , Saffari, Afshin (Author) ,
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Patients with Allan-Herndon-Dudley Syndrome (MCT8 deficiency) display symptoms of Parkinsonism in childhood and respond to Levodopa/Carbidopa treatment by Wilpert, Nina-Maria (Author) , Hewitt, Angela L. (Author) , Pons, Roser (Author) , Henke, Marie-Thérèse (Author) , Dell'Orco, Andrea (Author) , Bauer, Martin (Author) , Grolik, Christiane (Author) , Menz, Stephan (Author) , Wahle, Monika (Author) , Zink, Annika (Author) , Prigione, Alessandro (Author) , Reinauer, Christina (Author) , Lange, Catharina (Author) , Furth, Christian (Author) , Brockmann, Knut (Author) , Jung-Klawitter, Sabine (Author) , Christ, Stine (Author) , Kaindl, Angela M. (Author) , Tietze, Anna (Author) , Krude, Heiko (Author) , Opladen, Thomas (Author) , Schuelke, Markus (Author) ,
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3
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study by Weiß, Claudia (Author) , Becker, Lena-Luise (Author) , Friese, Johannes (Author) , Blaschek, Astrid (Author) , Hahn, Andreas (Author) , Illsinger, Sabine (Author) , Schwartz, Oliver (Author) , Bernert, Günther (Author) , Hagen, Maja von der (Author) , Husain, Ralf A. (Author) , Goldhahn, Klaus (Author) , Kirschner, Janbernd (Author) , Pechmann, Astrid (Author) , Flotats-Bastardas, Marina (Author) , Schreiber, Gudrun (Author) , Schara, Ulrike (Author) , Plecko, Barbara (Author) , Trollmann, Regina (Author) , Horber, Veronka (Author) , Wilichowski, Ekkehard (Author) , Baumann, Matthias (Author) , Klein, Andrea (Author) , Eisenkölbl, Astrid (Author) , Köhler, Cornelia (Author) , Stettner, Georg M. (Author) , Cirak, Sebahattin (Author) , Hasselmann, Oswald (Author) , Kaindl, Angela M. (Author) , Garbade, Sven (Author) , Johannsen, Jessika (Author) , Ziegler, Andreas (Author) ,
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4
β-Ureidopropionase deficiency due to novel and rare UPB1 mutations affecting pre-mRNA splicing and protein structural integrity and catalytic activity by Dobritzsch, Doreen (Author) , Meijer, Judith (Author) , Meinsma, Rutger (Author) , Maurer, Dirk (Author) , Monavari, Ardeshir A. (Author) , Gummesson, Anders (Author) , Reims, Annika (Author) , Cayuela, Jorge A. (Author) , Kuklina, Natalia (Author) , Benoist, Jean-François (Author) , Perrin, Laurence (Author) , Assmann, Birgit (Author) , Hoffmann, Georg F. (Author) , Bierau, Jörgen (Author) , Kaindl, Angela M. (Author) , van Kuilenburg, André B. P. (Author) ,
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5
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 by Weiß, Claudia (Author) , Ziegler, Andreas (Author) , Becker, Lena-Luise (Author) , Johannsen, Jessika (Author) , Brennenstuhl, Heiko (Author) , Schreiber, Gudrun (Author) , Flotats-Bastardas, Marina (Author) , Stoltenburg, Corinna (Author) , Hartmann, Hans (Author) , Illsinger, Sabine (Author) , Denecke, Jonas (Author) , Pechmann, Astrid (Author) , Müller-Felber, Wolfgang (Author) , Vill, Katharina (Author) , Blaschek, Astrid (Author) , Smitka, Martin (Author) , van der Stam, Lieske (Author) , Weiss, Katja (Author) , Winter, Benedikt (Author) , Goldhahn, Klaus (Author) , Plecko, Barbara (Author) , Horber, Veronka (Author) , Bernert, Günther (Author) , Husain, Ralf A (Author) , Rauscher, Christian (Author) , Trollmann, Regina (Author) , Garbade, Sven (Author) , Hahn, Andreas (Author) , von der Hagen, Maja (Author) , Kaindl, Angela M (Author) ,
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