التفاصيل البيبلوغرافية
العنوان:
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 trial
المؤلفون:
Eugenio Mercuri , Nicolas Deconinck , Elena S Mazzone , Andres Nascimento , Maryam Oskoui , Kayoko Saito , Carole Vuillerot , Giovanni Baranello , Odile Boespflug-Tanguy , Nathalie Goemans , Janbernd Kirschner , Anna Kostera-Pruszczyk , Laurent Servais , Marianne Gerber , Ksenija Gorni , Omar Khwaja , Heidemarie Kletzl , Renata S Scalco , Hannah Staunton , Wai Yin Yeung , Carmen Martin , Paulo Fontoura , John W Day , Joseph J. Volpe , John Posner , Ulrich Kellner , Rosaline Quinlivan , Aurore Daron , Stéphanie Delstanche , Romain Bruninx , Fabian Dal Farra , Olivier Schneider , Irina Balikova , Patricia Delbeke , Inge Joniau , Valentine Tahon , Sylvia Wittevrongel , Elke De Vos , Ingele Casteels , Liesbeth De Waele , Catherine Cassiman , Lies Prové , David Kinoo , Lisa Vancampenhout , Marleen Van Den Hauwe , Annelies Van Impe , Alexandra Prufer de Queiroz Campos Araujo , Aline Chacon Pereira , Flávia Nardes , Lorena Haefeli , Julia Rossetto , Marcos Ferreira Rebel , Jaqueline Almeida Pereira , Craig Campbell , Sapna Sharan , Wendy McDonald , Cheryl Scholtes , Jean Mah , Maria Sframeli , Angela Chiu , Jane Hagel , Raquel Beneish , Gaela Cariou-Palmer , Connie Pham , Daniela Toffoli , Stephanie Arpin , Sarah Turgeon Desilets , Yi Wang , Chaoping Hu , Jianfeng Huan , Chen Qian , 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 , Jingjing Zhao , Wenzhu Li , Lun Qin , Nina Barisic , Martina Galiot Delic , Petra Kristina Ivkic , Nenad Vukojevic , Ivana Kern , Boris Najdanovic , Marin Skugor , Teresa Gidaro , Andreea Seferian , Silvana De Lucia , Emmanuel Barreau , Nabila Mnafek , Marta Milkova Momtchilova , Helene Peche , Carole Valherie , Allison Grange , Charlotte Lilien , Darko Milascevic , Shotaro Tachibana , Claudia Ravelli , Ruxandra Cardas , Jessica Taytard , Guillaume Aubertin , Laure Vanden Brande , Jean-Baptiste Davion , Stephanie Coopman , Ikram Bouacha , Philippe Debruyne , Sabine Defoort , Gilles Derlyn , Florian Leroy , Loïc Danjoux , Julie Guilbaud , Isabelle Desguerre , Christine Barnérias , Michaela Semeraro , Dominique Bremond-Gignac , Lenaic Bruere , Maxence Rateaux , Élodie Deladrière , Virginie Germa , Yann Pereon , Sandra Mercie , Fanny Billaud , Lucie Le Goff , Guy Letellier , Aurélie Portefaix , Camille De-Montferrand , Laure Le-Goff , Stephanie Fontaine , Manel Saidi , Nabil Bouzid , Aurélie Barriere , Marie Tinat , Michelle Dreesbach , Wolf Lagréze , Bettina Michaelis , Fanni Molnar , Dorina Seger , Sibylle 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 Pietro Comi , Roberta Brusa , Stefania Corti , Velardo Daniele , Alessandra Govoni , Francesca Magri , Valeria Minorini , Silvia Gabriella Osnaghi , Francesca Abbati , Federica Fassini , Michaela Foa , Amaqlia Lopopolo , Megi Meneri , Francesca Zoppas , Valeria Parente , Riccardo Masson , Stefania Bianchi Marzoli , Diletta Santarsiero , Myriam Garcia Sierra , Gemma Tremolada , Maria Teresa Arnoldi , Marta Vigano , Riccardo Zanin , Laura Antonaci , Roberto de Sanctis , Marika Pane , Maria Carmela Pera , Giulia Maria Amorelli , Costanza Barresi , Gugliemo 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 , Kenishi Fudeyasu , Kazuhiko Hirata , Kana Michiue , Kazuyuki Ueda , 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 Fraczek , Maria Jedrzejowska , Anna Lusakowska , Agnieszka Czeszyk-Piotrowicz , Wojciech Hautz , Klaudia Rakusiewicz , Malgorzata Burlewicz , Zuzanna Gierlak-Wojcicka , Malwina Kepa , Adam Sikorski , Marcin Sobieraj , Maria Mazurkiewicz-Beldzinska , Anna Lemska , Sandra Modrzejewska , Mateusz Koberda , Urszula Stodolska-Koberda , Agnieszka Waskowska , Jagoda Kolendo , Agnieszka Sobierajska-Rek , Barbara Steinborn , Magdalena Dalz , Julia Grabowska , Wojciech Hajduk , Justyna Janasiewicz-Karachitos , Monika Klimas , Marcin Stopa , Ewa Gajewska , Beata Pusz , Dmitry Vlodavets , Evgenia Melnik , Natalya Leppenen , Nataliya Yupatova , Anastasya Monakhova , Yulia Papina , Olga Shidlovsckaia , Vedrana Milic Rasic , Vesna Brankovic , Ana Kosac , Olivera Djokic , Vesna Jakšic , Ana Pepic , Jelena Martinovic , Francina Munell Casadesus , Eduardo Tizzano , Nieves Martín Begué , Charlotte Wolley Dod , Olaia Subira , Bernat Planas Pascual , Esther Toro Tamargo , Marcos Madruga Garrido , José David Medina Romero , Marta Peña Salinas , Andrés Nascimento Osorio , Ana Díaz Cortés , Enrique Jiménez Gañan , Simone Dowon Suh , Julita Medina Cantillo , Obdulia Moya , Nuria Padros , Sandra Roca Urraca , Hugo Gonzalez Valdivia , Samuel Pascual Pascual , Sofía de Manuel , Susana Noval Martin , Paul Burnham , Sandra Espinosa , Mercedes Martinez Moreno , Haluk Topaloglu , Ibrahim Oncel , Nesibe Eroglu Ertugru , Bahadir Konuskan , Bora Eldem , Sibel Kadayifçilar , Ipek Alemdaroglu , Aynur Ayse Karaduman , Oznur Tunca Yilmaz , Neslihan Bilgin , Seher Sari , Claudia Chiriboga , John J. Lee , Donnielle Rome-Martin , John W. Day , Shannon Beres , Tina Duong , Richard Gee , Sally Dunaway Young , Sabine Fuerst-Recktenwald , Anne Marquet , Nicoletta Muelhardt , Dylan Trundell
المصدر:
LANCET NEUROLOGY r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu instname
بيانات النشر:
Elsevier BV, 2022.
سنة النشر:
2022
مصطلحات موضوعية:
Adult , Risdiplam , spinal muscular atrophy , Adolescent , Spinal Muscular Atrophies of Childhood , Settore MED/26 - NEUROLOGIA , Young Adult , Pyrimidines , Settore MED/39 - NEUROPSICHIATRIA INFANTILE , Double-Blind Method , Child, Preschool , Humans , Neurology (clinical) , Child , Preschool , Azo Compounds , Aged
الوصف:
BACKGROUND: 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. METHODS: In this phase 3, randomised, double-blind, placebo-controlled study, patients aged 2-25 years with confirmed 5q autosomal recessive type 2 or type 3 spinal muscular atrophy were recruited from 42 hospitals in 14 countries across Europe, North America, South America, and Asia. Participants were eligible if they were non-ambulant, could sit independently, and had a score of at least 2 in entry item A of the Revised Upper Limb Module. Patients were stratified by age and randomly assigned (2:1) to receive either daily oral risdiplam, at a dose of 5·00 mg (for individuals weighing =20 kg) or 0·25 mg/kg (for individuals weighing
تدمد:
1474-4422
الوصول الحر:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::917024073c62a8e3c3c8eedaa6f463bbTest https://doi.org/10.1016/s1474-4422Test (21)00367-7
حقوق:
OPEN
رقم الانضمام:
edsair.doi.dedup.....917024073c62a8e3c3c8eedaa6f463bb
قاعدة البيانات:
OpenAIRE