1
TITLE: The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
AUTHORS: Palomo, Gloria M.; Pose Boirazian, Tomas; Naumann Winter, Frauke; Costa, Enrico; Duarte, Dinah M.; Kalland, Maria E.; Malikova, Eva; Matusevicius, Darius; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova Beninska, Violeta; Mariz, Segundo;
PUBLISHED: 2023, SOURCE: MOLECULAR THERAPY, VOLUME: 31, ISSUE: 12
INDEXED IN: WOS
2
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
3
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
4
TITLE: Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union  Full Text
AUTHORS: Stelios Tsigkos; Segundo Mariz; Jordi Llinares; Laura Fregonese; Stiina Aarum; Frauke Naumann-Winter; Kerstin Westermark; Bruno Sepodes;
PUBLISHED: 2015, SOURCE: Orphanet Journal of Rare Diseases - Orphanet J Rare Dis, VOLUME: 10, ISSUE: 1
INDEXED IN: CrossRef
5
TITLE: Establishing medical plausibility in the context of orphan medicines designation in the European Union (vol 9, 175, 2014)  Full Text
AUTHORS: Stelios Tsigkos; Segundo Mariz; Jordi Llinares; Laura Fregonese; Stiina Aarum; Frauke Naumann Winter; Kerstin Westermark; Bruno Sepodes;
PUBLISHED: 2015, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 10
INDEXED IN: WOS
6
TITLE: Use of biomarkers in the context of orphan medicines designation in the European Union  Full Text
AUTHORS: Stelios Tsigkos; Jordi Llinares; Segundo Mariz; Stiina Aarum; Laura Fregonese; Bozenna Dembowska Baginska; Rembert Elbers; Pauline Evers; Tatiana Foltanova; Andre Lhoir; Ana Correa Nunes; Daniel O'Connor; Albertha Voordouw; Kerstin Westermark; Bruno Sepodes;
PUBLISHED: 2014, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 9, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
7
TITLE: The orphan framework as a new opportunity: an expert opinion
AUTHORS: Segundo Mariz; Stelios Tsigkos; Laura Fregonese; Stiina Aarum; Eleonora Dehlink; Jordi Llinares; Bruno Sepodes;
PUBLISHED: 2014, SOURCE: EXPERT OPINION ON ORPHAN DRUGS, VOLUME: 2, ISSUE: 11
INDEXED IN: Scopus WOS CrossRef
8
TITLE: Establishing medical plausibility in the context of orphan medicines designation in the European Union  Full Text
AUTHORS: Stelios Tsigkos; Segundo Mariz; Jordi Llinares; Laura Fregonese; Stiina Aarum; Naumann Winter Frauke; Kerstin Westermark; Bruno Sepodes;
PUBLISHED: 2014, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 9, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef