ProfileOfResearchers

TITLE: Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy Full Text
AUTHORS: 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; ...More
PUBLISHED: 2016, SOURCE: LANCET NEUROLOGY, VOLUME: 15, ISSUE: 8

INDEXED IN:

Scopus

WOS

TITLE: The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations Full Text
AUTHORS: 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; ...More
PUBLISHED: 2015, SOURCE: HUMAN MUTATION, VOLUME: 36, ISSUE: 4

INDEXED IN:

Scopus

WOS

Useful Links