Eugenio Mercuri, Nicolas Deconinck, Elena Mazzone, A. Nascimento, Maryam Oskoui, Kayoko Saito, Carole Vuillerot, Giovanni Baranello, Odile Boespflüg-Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera‐Pruszczyk, Laurent Servais, Marianne Gerber, Ksenija Gorni, Omar Khwaja, Heidemarie Kletzl, R. Scalco, Hannah Staunton, Wai Yin Yeung, Carmen Martín, Paulo Fontoura, John Day, Joseph J. Volpe, John Posner, Ulrich Kellner, Rosaline C. M. Quinlivan, Aurore Daron, Stéphanie Delstanche, Romain Bruninx, Fabian Dal Farra, O. Schneider, Nicolas Deconinck, Irina Balikova, Patricia Delbeke, Inge Joniau, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Nathalie Goemans, Ingele Casteels, Liesbeth De Waele, Irina Balikova, Cathérine Cassiman, Lies Prové, David Kinoo, Lisa Vancampenhout, M. van den Hauwe, Annelies Van Impe, Alexandra Prufer de Queiroz Campos Araújo, Aline Chacon Pereira, Flávia Nardes, Lorena Haefeli, Júlia Dutra Rossetto, Marcos Ferreira Rebel, Jaqueline Almeida Pereira, Craig Campbell, Sapna Sharan, Wendy L. McDonald, Cheryl Scholtes, Jean K. Mah, Maria Sframeli, Angela W. Chiu, Jane Hagel, Maryam Oskoui, Raquel Beneish, Gaela Cariou-Palmer, Connie Pham, Daniela Toffoli, Stéphanie Arpin, Sarah Turgeon Desilets, Yi Wang, Chaoping Hu, Jianfeng Huan, Qian Chen, Li Shen, Ying Xiao, Zhenxuan Zhou, Hui Li, Sujuan Wang, Hui Xiong, Xingzhi Chang, Hui Dong, Ying Liu, Tian Sang, Cuijie Wei, Jing Wen, Yiwen Cao, Xingyao Ly, Jing Wen, Jingjing Zhao, Wenzhu Li, Lun Qin, Nina Barišić, Martina Galiot Delić, Petra Kristina Ivkić, Nenad Vukojević, Ivana Kern, B Najdanović, Marin Skugor, Laurent Servais, Odile Boespflüg-Tanguy, Teresa Gidaro, A. Seferian, Silvana De Lucia, Emmanuel Barreau, Nabila Mnafek, Marta Momtchilova, Helene Peche, Carole Valherie, Allison Grange, Charlotte Lilien, Darko Milaščević, Shotaro Tachibana, Claudia Ravelli, Ruxandra Cardaş, Jessica Taytard, Guillaume Aubertin, Laure Vanden Brande, Jean‐Baptiste Davion, Stéphanie Coopman, Ikram Bouacha, Philippe Debruyne, Sabine Defoort, Gilles Derlyn, Florian Leroy, Loïc Danjoux, J Guilbaud, Isabelle Desguerre, Christine Barnérias, Michaëla Semeraro, Dominique Brémond‐Gignac, Lenaïc Bruère, Maxence Rateaux, Élodie Deladrière, Virginie Germa, Yann Péréon, Sandra Mercie, Fanny Billaud, Lucie Le Goff, Guy Letellier, Carole Vuillerot, Aurélie Portefaix, Camille De-Montferrand, Laure Le-Goff, Stéphanie Fontaine, Manel Saidi, N. Bouzid, Aurélie Barrière, Marie Tinat, Janbernd Kirschner, Michelle Dreesbach, Wolf A. Lagrèze, Bettina Michaelis, Fanni Molnár, Dorina Seger, Sibylle Emilie Vogt, Enrico Bertini, Adele D’Amico, Sergio Petroni, Anna Maria Bonetti, Adelina Carlesi, Irene Mizzoni, Claudio Bruno, Enrico Priolo, Giuseppe Rao, Simone Morando, Paola Tacchetti, Ambra Zuffi, Giacomo P. Comi, Roberta Brusa, Stefania Corti, Velardo Daniele, Alessandra Govoni, Francesca Magri, Valeria Minorini, Silvia Osnaghi, Francesca Abbati, Federica Fassini, Michaela Foà, Amaqlia Lopopolo, Megi Meneri, Francesca Zoppas, Valeria Parente, Giovanni Baranello, Riccardo Masson, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, M. E. F Viganó, Riccardo Zanin, Eugenio Mercuri, Laura Antonaci, Roberto De Sanctis, Marika Pane, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, G. D'Amico, Lorenzo Orazi, Giorgia Coratti, Kazuhiro Haginoya, Atsuko Kato, Yuko Morishita, Ryutaro Kira, Kiyomu Akiyama, Miwako Goto, Yujiro Mori, Misato Okamoto, Saki Tsutsui, Yuta Takatsuji, Aya Tanaka, Hirofumi Komaki, Miina Omori, Ippei Suzuki, Mizuki Takeuchi, Daisuke Todoroki, Seji Watanabe, Tomoko Matsubayashi, Emi Inakazu, Hiroe Nagura, Akira Suzuki, Manami Usui, Nobutsune Ishikawa, Yousuke Harada, Kenichi Fudeyasu, Kazuhiko Hirata, Kana Michiue, Kazuyuki Ueda, Kayoko Saito, Junko Fujitani, Reiko Arakawa, Kozue Takano, Shigeko Yashiro, Maiko Seki, Nozomi Sano, Koji Fukuyama, Yuki Matsumoto, Hirofumi Miyazaki, Minoru Shibata, Kyoko Kobayashi, Yukie Nakamura, Yasuhiro Takeshima, Moe Kuma, Anna Kostera‐Pruszczyk, Anna Frączek, Maria Jędrzejowska, Anna Łusakowska, Agnieszka Czeszyk-Piotrowicz, Wojciech Hautz, Klaudia Rakusiewicz, Małgorzata Burlewicz, Zuzanna Gierlak-Wojcicka, Malwina Kępa, Adam Sikorski, Marcin Sobieraj, Maria Mazurkiewicz-Bełdzińska, Anna Lemska, Sandra Modrzejewska, Mateusz Koberda, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Barbara Steinborn, Magdalena Dalz, J Grabowska, Wojciech Hajduk, Justyna Janasiewicz-Karachitos, Monika Klimas, Marcin Stopa, Ewa Gajewska, Beata Pusz, D. Vlodavets, Evgenia Melnik, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Vedrana Milić Rašić, V. Branković, Ana Kosać, Olivera Djokić, Vesna Jakšić, Ana Pepić, Jelena Martinović, Francina Munell, Eduardo F. Tizzano, Nieves Martín Begué, Charlotte Wolley-Dod, Olaia Subirà, Bernat Planas Pascual, Esther Toro-Tamargo,
Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy.In this phase 3, randomised, double-blind, placebo- ...
Elsevier BV