Real-World Data from Nusinersen Treatment for Patients with Later-Onset Spinal Muscular Atrophy: A Single Center Experience von Mendonça, Rodrigo H., Polido, Graziela J., Matsui, Ciro, Silva, André M.S., Solla, Davi J.F., Reed, Umbertina C., Zanoteli, Edmar

Volltext

Risdiplam in Type 1 Spinal Muscular Atrophy von Baranello, Giovanni, Darras, Basil T, Day, John W, Deconinck, Nicolas, Klein, Andrea, Masson, Riccardo, Mercuri, Eugenio, Rose, Kristy, El-Khairi, Muna, Gerber, Marianne, Gorni, Ksenija, Khwaja, Omar, Kletzl, Heidemarie, Scalco, Renata S, Seabrook, Timothy, Fontoura, Paulo, Servais, Laurent

Volltext

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

Volltext