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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: Aartsma Rus, A; Straub, V; Hemmings, R; Haas, M; Schlosser Weber, G; Stoyanova Beninska, V; Mercuri, E; Muntoni, F; Sepodes, B; Vroom, E; Balabanov, P;
PUBLISHED: 2017, SOURCE: NUCLEIC ACID THERAPEUTICS, VOLUME: 27, ISSUE: 5
INDEXED IN: Scopus WOS CrossRef: 141
IN MY: ORCID
72
TITLE: Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework
AUTHORS: Carvalho, M; Sepodes, B; Martins, AP;
PUBLISHED: 2017, SOURCE: FRONTIERS IN MEDICINE, VOLUME: 4, ISSUE: OCT
INDEXED IN: Scopus WOS CrossRef: 41
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73
TITLE: Advanced therapy medicinal products for rare diseases: State of play of incentives supporting development in Europe
AUTHORS: Andreas M Farkas; Segundo Mariz; Violeta Stoyanova Beninska; Patrick Celis; Spiros Vamvakas; Kristina Larsson; Bruno Sepodes;
PUBLISHED: 2017, SOURCE: Frontiers in Medicine, VOLUME: 4, ISSUE: MAY
INDEXED IN: Scopus CrossRef: 24
IN MY: ORCID
74
TITLE: Red Raspberry Phenols Inhibit Angiogenesis: A Morphological and Subcellular Analysis Upon Human Endothelial Cells. RASPBERRYPOLYPHENOLSInANGIOGENESIS  Full Text
AUTHORS: Sousa, M; Machado, V ; Costa, R; E. Figueira; Sepodes, B; Barata, P ; Ribeiro, L ; Soares, R ;
PUBLISHED: 2016, SOURCE: JOURNAL OF CELLULAR BIOCHEMISTRY, VOLUME: 117, ISSUE: 7
INDEXED IN: Scopus WOS CrossRef: 13
IN MY: ORCID
75
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
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76
TITLE: Regulatory watch: The orphan drug pipeline in Europe  Full Text
AUTHORS: Morel, T; Lhoir, A; Picavet, E; Mariz, S; Sepodes, B; Llinares, J; Cassiman, D;
PUBLISHED: 2016, SOURCE: Nature Reviews Drug Discovery, VOLUME: 15, ISSUE: 6
INDEXED IN: Scopus CrossRef
IN MY: ORCID
77
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
IN MY: ORCID
78
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
IN MY: ORCID
80
TITLE: Worldwide collaboration for orphan drug designation (vol 15 , pg 440, 2016)  Full Text
AUTHORS: Mariz, S; Reese, JH; Westermark, K; Greene, L; Goto, T; Hoshino, T; Llinares Garcia, J; Sepodes, B;
PUBLISHED: 2016, SOURCE: NATURE REVIEWS DRUG DISCOVERY, VOLUME: 15, ISSUE: 8
INDEXED IN: WOS
IN MY: ORCID
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