Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Author:

Masson Riccardo,Mazurkiewicz-Bełdzińska Maria,Rose Kristy,Servais Laurent,Xiong Hui,Zanoteli Edmar,Baranello Giovanni,Bruno Claudio,Day John W,Deconinck Nicolas,Klein Andrea,Mercuri Eugenio,Vlodavets Dmitry,Wang Yi,Dodman Angela,El-Khairi Muna,Gorni Ksenija,Jaber Birgit,Kletzl Heidemarie,Gaki Eleni,Fontoura Paulo,Darras Basil T,Volpe Joseph J,Posner John,Kellner Ulrich,Quinlivan Rosaline,Gerber Marianne,Khwaja Omar,Scalco Renata S,Seabrook Timothy,Koch Armin,Balikova Irina,Joniau Inge,Accou Geraldine,Tahon Valentine,Wittevrongel Sylvia,De Vos Elke,de Holanda Mendonça Rodrigo,Matsui Jr Ciro,Fornazieri Darcie Ana Letícia,Machado Cleide,Kiyoko Oyamada Maria,Martini Joyce,Polido Graziela,Rodrigues Iannicelli Juliana,Caires de Oliveira Achili Ferreira Juliana,Hu Chaoping,Zhu Xiaomei,Qian Chen,Shen Li,Li Hui,Shi Yiyun,Zhou Shuizhen,Xiao Ying,Zhou Zhenxuan,Wang Sujuan,Sang Tian,Wei Cuijie,Dong Hui,Cao Yiwen,Wen Jing,Li Wenzhu,Qin Lun,Barisic Nina,Celovec Ivan,Galiot Delic Martina,Ivkic Petra Kristina,Vukojevic Nenad,Kern Ivana,Najdanovic Boris,Skugor Marin,Tomas Josipa,Boespflug-Tanguy Odile,De Lucia Silvana,Seferian Andrea,Barreau Emmanuel,Mnafek Nabila,Peche Helene,Grange Allison,Trang Nguyen Diem,Milascevic Darko,Tachibana Shotaro,Pagliano Emanuela,Bianchi Marzoli Stefania,Santarsiero Diletta,Garcia Sierra Myriam,Tremolada Gemma,Arnoldi Maria Teresa,Vigano Marta,Dosi Claudia,Zanin Riccardo,Schembri Veronica,Brolatti Noemi,Rao Giuseppe,Tassara Elisa,Morando Simone,Tacchetti Paola,Pedemonte Marina,Priolo Enrico,Sposetti Lorenza,Comi Giacomo Pietro,Govoni Alessandra,Osnaghi Silvia Gabriella,Minorini Valeria,Abbati Francesca,Fassini Federica,Foa Michaela,Lopopolo Amalia,Pane Marika,Palermo Concetta,Pera Maria Carmela,Amorelli Giulia Maria,Barresi Costanza,D'Amico Guglielmo,Orazi Lorenzo,Coratti Giorgia,Leone Daniela,Laura Antonaci,De Sanctis Roberto,Berti Beatrice,Kimura Naoki,Takeshima Yasuhiro,Shimomura Hideki,Lee Tomoko,Gomi Fumi,Morimatsu Takanobu,Furukawa Toru,Stodolska-Koberda Urszula,Waskowska Agnieszka,Kolendo Jagoda,Sobierajska-Rek Agnieszka,Modrzejewska Sandra,Lemska Anna,Melnik Evgenia,Artemyeva Svetlana,Leppenen Natalya,Yupatova Nataliya,Monakhova Anastasya,Papina Yulia,Shidlovsckaia Olga,Litvinova Elena,Enzmann Cornelia,Galiart Elea,Gugleta Konstantin,Wondrusch Haschke Christine,Topaloglu Haluk,Oncel Ibrahim,Ertugrul Nesibe Eroglu,Konuskan Bahadir,Eldem Bora,Kadayifçilar Sibel,Alemdaroglu Ipek,Sari Seher,Bilgin Neslihan,Karaduman Aynur Ayse,Sarikaya Fatma Gokcem Yildiz,Graham Robert J,Ghosh Partha,Casavant David,Levine Alexis,Titus Rachael,Engelbrekt Amanda,Ambrosio Lucia,Fulton Anne,Baglieri Anna Maria,Dias Courtney,Maczek Elizabeth,Pasternak Amy,Beres Shannon,Duong Tina,Gee Richard,Young Sally

Publisher

Elsevier BV

Subject

Neurology (clinical)

Reference28 articles.

1. Identification and characterization of a spinal muscular atrophy-determining gene;Lefebvre;Cell,1995

2. Swaiman's Pediatric Neurology: principles and practice;Darras,2017

3. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy;Lorson;Proc Natl Acad Sci USA,1999

4. Diverse role of survival motor neuron protein;Singh;Biochim Biophys Acta Gene Regul Mech,2017

5. Natural history of infantile-onset spinal muscular atrophy;Kolb;Ann Neurol,2017

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