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    Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled tria... von Nascimento, Andres, Vuillerot, Carole, Boespflug-Tanguy, Odile, Kirschner, Janbernd, Kostera-Pruszczyk, Anna, Kletzl, Heidemarie, Day, John W, Volpe, Joseph J., Posner, John, Daron, Aurore, Delbeke, Patricia, Wittevrongel, Sylvia, De Vos, Elke, De Waele, Liesbeth, Prufer de Queiroz Campos Araujo, Alexandra, Haefeli, Lorena, Chiu, Angela, Oskoui, Maryam, Toffoli, Daniela, Turgeon Desilets, Sarah, Wang, Yi, Shen, Li, Li, Hui, Sang, Tian, Barisic, Nina, Galiot Delic, Martina, Ivkic, Petra Kristina, Kern, Ivana, Momtchilova, Marta Milkova, Milascevic, Darko, Aubertin, Guillaume, Coopman, Stephanie, Debruyne, Philippe, Derlyn, Gilles, Danjoux, Loïc, Deladrière, Élodie, Germa, Virginie, Billaud, Fanny, Le Goff, Lucie, Letellier, Guy, De-Montferrand, Camille, Le-Goff, Laure, Fontaine, Stephanie, Kirschner, Janbernd, Lagréze, Wolf, Bertini, Enrico, D'Amico, Adele, Carlesi, Adelina, Zuffi, Ambra, Comi, Giacomo Pietro, Daniele, Velardo, Minorini, Valeria, Fassini, Federica, Foa, Michaela, Parente, Valeria, Masson, Riccardo, Tremolada, Gemma, Amorelli, Giulia Maria, Orazi, Lorenzo, Coratti, Giorgia, Morishita, Yuko, Goto, Miwako, Tsutsui, Saki, Takatsuji, Yuta, Komaki, Hirofumi, Omori, Miina, Nagura, Hiroe, Usui, Manami, Fudeyasu, Kenishi, Hirata, Kazuhiko, Michiue, Kana, Fujitani, Junko, Seki, Maiko, Fukuyama, Koji, Shibata, Minoru, Kobayashi, Kyoko, Nakamura, Yukie, Sikorski, Adam, Koberda, Mateusz, Stodolska-Koberda, Urszula, Waskowska, Agnieszka, Kolendo, Jagoda, Steinborn, Barbara, Klimas, Monika, Stopa, Marcin, Gajewska, Ewa, Leppenen, Natalya, Brankovic, Vesna, Djokic, Olivera, Planas Pascual, Bernat, Salinas, Marta Peña, Padros, Nuria, Oncel, Ibrahim, Eroglu Ertugru, Nesibe, Kadayifçilar, Sibel, Karaduman, Aynur Ayse, Chiriboga, Claudia, Marquet, Anne, Muelhardt, Nicoletta, Trundell, Dylan

    Veröffentlicht in Lancet neurology

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    Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial von Masson, Riccardo, Mazurkiewicz-Bełdzińska, Maria, Xiong, Hui, Bruno, Claudio, Day, John W, Deconinck, Nicolas, Klein, Andrea, Mercuri, Eugenio, Wang, Yi, Dodman, Angela, Gaki, Eleni, Posner, John, Kellner, Ulrich, Quinlivan, Rosaline, Khwaja, Omar, Scalco, Renata S, Seabrook, Timothy, Koch, Armin, Balikova, Irina, Tahon, Valentine, De Vos, Elke, de Holanda Mendonça, Rodrigo, Machado, Cleide, Polido, Graziela, Hu, Chaoping, Qian, Chen, Li, Hui, Zhou, Shuizhen, Zhou, Zhenxuan, Wei, Cuijie, Dong, Hui, Wen, Jing, Qin, Lun, Celovec, Ivan, Galiot Delic, Martina, Ivkic, Petra Kristina, Vukojevic, Nenad, Kern, Ivana, Boespflug-Tanguy, Odile, De Lucia, Silvana, Barreau, Emmanuel, Mnafek, Nabila, Peche, Helene, Pagliano, Emanuela, Bianchi Marzoli, Stefania, Santarsiero, Diletta, Tremolada, Gemma, Vigano, Marta, Dosi, Claudia, Zanin, Riccardo, Schembri, Veronica, Brolatti, Noemi, Rao, Giuseppe, Tassara, Elisa, Sposetti, Lorenza, Govoni, Alessandra, Osnaghi, Silvia Gabriella, Minorini, Valeria, Abbati, Francesca, Fassini, Federica, Foa, Michaela, Lopopolo, Amalia, Pane, Marika, Palermo, Concetta, Barresi, Costanza, Orazi, Lorenzo, Laura, Antonaci, De Sanctis, Roberto, Berti, Beatrice, Kimura, Naoki, Shimomura, Hideki, Lee, Tomoko, Morimatsu, Takanobu, Waskowska, Agnieszka, Kolendo, Jagoda, Sobierajska-Rek, Agnieszka, Modrzejewska, Sandra, Lemska, Anna, Melnik, Evgenia, Leppenen, Natalya, Papina, Yulia, Shidlovsckaia, Olga, Enzmann, Cornelia, Wondrusch Haschke, Christine, Topaloglu, Haluk, Oncel, Ibrahim, Ertugrul, Nesibe Eroglu, Konuskan, Bahadir, Alemdaroglu, Ipek, Sari, Seher, Karaduman, Aynur Ayse, Graham, Robert J, Ghosh, Partha, Levine, Alexis, Engelbrekt, Amanda, Ambrosio, Lucia, Baglieri, Anna Maria, Maczek, Elizabeth, Duong, Tina, Young, Sally

    Veröffentlicht in Lancet neurology

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