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TÍTULO: Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy  Full Text
AUTORES: Volker Straub; Pavel Balabanov; Kate Bushby; Monica Ensini; Nathalie Goemans; Annamaria De Luca; Alejandra Pereda; Robert Hemmings; Giles Campion; Edward Kaye; Virginia Arechavala Gomeza; Aurelie Goyenvalle; Erik Niks; Olav Veldhuizen; Pat Furlong; Violeta Stoyanova Beninska; Matthew J Wood; Alex Johnson; Eugenio Mercuri; Francesco Muntoni; Bruno Sepodes; Manuel Haas; Elizabeth Vroom; Annemieke Aartsma Rus; ...Mais
PUBLICAÇÃO: 2016, FONTE: LANCET NEUROLOGY, VOLUME: 15, NÚMERO: 8
INDEXADO EM: Scopus WOS CrossRef
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TÍTULO: The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations  Full Text
AUTORES: Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barisic; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaida Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera Pruszczyk; Janusz Zimowski; Rosario Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre Yves Jeannet; Franziska Joncourt; Jordi Diaz Manera; Eduard Gallardo; Ayse A Karaduman; Haluk Topaloglu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma Rus; Christophe Beroud; Hanns Lochmueller; ...Mais
PUBLICAÇÃO: 2015, FONTE: HUMAN MUTATION, VOLUME: 36, NÚMERO: 4
INDEXADO EM: Scopus WOS CrossRef