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TITLE: Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
AUTHORS: Annemieke Aartsma Rus; Volker Straub; Robert Hemmings; Manuel Haas; Gabriele Schlosser Weber; Violeta Stoyanova Beninska; Eugenio Mercuri; Francesco Muntoni; Bruno Sepodes; Elizabeth Vroom; Pavel Balabanov;
PUBLISHED: 2017, SOURCE: NUCLEIC ACID THERAPEUTICS, VOLUME: 27, ISSUE: 5
INDEXED IN: Scopus WOS
43
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 CrossRef
44
TITLE: The orphan drug pipeline in Europe
AUTHORS: Thomas Morel; Eline Picavet; Andre Lhoir; Jordi Llinares; Segundo Mariz; Bruno Sepodes; David Cassiman;
PUBLISHED: 2016, SOURCE: NATURE REVIEWS DRUG DISCOVERY, VOLUME: 15, ISSUE: 6
INDEXED IN: WOS
45
TITLE: Worldwide collaboration for orphan drug designation
AUTHORS: Segundo Mariz; James H Reese; Kerstin Westermark; Lesley Greene; Takahiro Goto; Tatsuro Hoshino; Jordi Llinares Garcia; Bruno Sepodes;
PUBLISHED: 2016, SOURCE: NATURE REVIEWS DRUG DISCOVERY, VOLUME: 15, ISSUE: 6
INDEXED IN: Scopus WOS CrossRef
47
TITLE: Protective effects of hydroxytyrosol-supplemented refined olive oil in animal models of acute inflammation and rheumatoid arthritis  Full Text
AUTHORS: Silva, S; Sepodes, B; Rocha, J; Direito, R; Fernandes, A; Brites, D; Freitas, M; Fernandes, E ; Bronze, MR; Figueira, ME;
PUBLISHED: 2015, SOURCE: JOURNAL OF NUTRITIONAL BIOCHEMISTRY, VOLUME: 26, ISSUE: 4
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
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