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: Defining orphan conditions in the context of the European orphan regulation: challenges and evolution
AUTHORS: Daniel J O'Connor; Maria E Sheean; Matthias P Hofer; Stelios Tsigkos; Segundo Mariz; Laura Fregonese; Kristina Larsson; Virginie Hivert; Kerstin Westermark; Frauke Naumann Winter; Violeta Stoyanova Beninska; Ingeborg Barisic; Giuseppe Capovilla; Armando Magrelli; Bruno Sepodes;
PUBLISHED: 2019, SOURCE: NATURE REVIEWS DRUG DISCOVERY, VOLUME: 18, ISSUE: 7
INDEXED IN: WOS
3
TITLE: Establishing rarity in the context of orphan medicinal product designation in the European Union  Full Text
AUTHORS: Stelios Tsigkos; Matthias Philipp Hofer; Maria Elzbieta Sheean; Segundo Mariz; Kristina Larsson; Frauke Naumann Winter; Laura Fregonese; Bruno Sepodes;
PUBLISHED: 2018, SOURCE: DRUG DISCOVERY TODAY, VOLUME: 23, ISSUE: 3
INDEXED IN: WOS
4
TITLE: Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations
AUTHORS: Andrea Ecker; Segundo Mariz; Frauke Naumann Winter; Koenraad Norga; Ingeborg Barisic; Thomas Girard; Paolo Tomasi; Dirk Mentzer; Bruno Sepodes;
PUBLISHED: 2018, SOURCE: ARCHIVES OF DISEASE IN CHILDHOOD, VOLUME: 103, ISSUE: 5
INDEXED IN: Scopus WOS
5
TITLE: Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union  Full Text
AUTHORS: Benedetta Polsinelli; Stelios Tsigkos; Frauke Naumann Winter; Segundo Mariz; Bruno Sepodes;
PUBLISHED: 2017, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 12, ISSUE: 1
INDEXED IN: Scopus WOS
6
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
7
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