1
TITLE: Real-World Evidence to Support EU Regulatory Decision Making-Results From a Pilot of Regulatory Use Cases
AUTHORS: Prilla, Stefanie; Groeneveld, Sophie; Pacurariu, Alexandra; Restrepo Mendez, Maria Clara; Verpillat, Patrice; Torre, Carla; Gartner, Christian; Mol, Peter G. M.; Naumann Winter, Frauke; Breen, Kieran C.; Gault, Nathalie; Gross Martirosyan, Liana; Benchetrit, Sylvie; Aylward, Brian; Stoyanova Beninska, Violeta; O'Donovan, Maura; Straus, Sabine; Kjaer, Jesper; Arlett, Peter;
PUBLISHED: 2024, SOURCE: CLINICAL PHARMACOLOGY & THERAPEUTICS
INDEXED IN: Scopus WOS
2
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
3
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
4
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