201 related articles for article (PubMed ID: 25475155)
1. Establishing medical plausibility in the context of orphan medicines designation in the European Union.
Tsigkos S; Mariz S; Llinares J; Fregonese L; Aarum S; Naumann-Winter F; Westermark K; Sepodes B
Orphanet J Rare Dis; 2014 Dec; 9():175. PubMed ID: 25475155
[TBL] [Abstract][Full Text] [Related]
2. Establishing rarity in the context of orphan medicinal product designation in the European Union.
Tsigkos S; Hofer MP; Sheean ME; Mariz S; Larsson K; Naumann-Winter F; Fregonese L; Sepodes B
Drug Discov Today; 2018 Mar; 23(3):681-686. PubMed ID: 28647377
[TBL] [Abstract][Full Text] [Related]
3. Use of biomarkers in the context of orphan medicines designation in the European Union.
Tsigkos S; Llinares J; Mariz S; Aarum S; Fregonese L; Dembowska-Baginska B; Elbers R; Evers P; Foltanova T; Lhoir A; Corrêa-Nunes A; O'Connor D; Voordouw A; Westermark K; Sepodes B
Orphanet J Rare Dis; 2014 Jan; 9():13. PubMed ID: 24461084
[TBL] [Abstract][Full Text] [Related]
4. The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products.
Palomo GM; Pose-Boirazian T; Naumann-Winter F; Costa E; Duarte DM; Kalland ME; Malikova E; Matusevicius D; Vitezic D; Larsson K; Magrelli A; Stoyanova-Beninska V; Mariz S
Mol Ther; 2023 Dec; 31(12):3414-3423. PubMed ID: 37794679
[TBL] [Abstract][Full Text] [Related]
5. Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.
Pauwels K; Huys I; Casteels M; Larsson K; Voltz C; Penttila K; Morel T; Simoens S
Orphanet J Rare Dis; 2017 Feb; 12(1):36. PubMed ID: 28209180
[TBL] [Abstract][Full Text] [Related]
6. Drug development for exceptionally rare metabolic diseases: challenging but not impossible.
Putzeist M; Mantel-Teeuwisse AK; Wied CC; Hoes AW; Leufkens HG; de Vrueh RL
Orphanet J Rare Dis; 2013 Nov; 8():179. PubMed ID: 24237580
[TBL] [Abstract][Full Text] [Related]
7. [European incentives for orphan medicinal products].
Enzmann H; Lütz J
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz; 2008 May; 51(5):500-8. PubMed ID: 18696141
[TBL] [Abstract][Full Text] [Related]
8. [Hope for patients with rare diseases--"orphan" drugs].
Kuzelová M; Kubácková K; Palágyi M; Smíd M
Cas Lek Cesk; 2006; 145(4):296-300. PubMed ID: 16639930
[TBL] [Abstract][Full Text] [Related]
9. Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union.
Polsinelli B; Tsigkos S; Naumann-Winter F; Mariz S; Sepodes B
Orphanet J Rare Dis; 2017 Jan; 12(1):17. PubMed ID: 28109318
[TBL] [Abstract][Full Text] [Related]
10. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
Joppi R; Bertele' V; Garattini S
Eur J Clin Pharmacol; 2013 Apr; 69(4):1009-24. PubMed ID: 23090701
[TBL] [Abstract][Full Text] [Related]
11. Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs.
Giannuzzi V; Landi A; Bosone E; Giannuzzi F; Nicotri S; Torrent-Farnell J; Bonifazi F; Felisi M; Bonifazi D; Ceci A
BMJ Open; 2017 Sep; 7(9):e017358. PubMed ID: 28893754
[TBL] [Abstract][Full Text] [Related]
12. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
Giannuzzi V; Conte R; Landi A; Ottomano SA; Bonifazi D; Baiardi P; Bonifazi F; Ceci A
Orphanet J Rare Dis; 2017 Apr; 12(1):64. PubMed ID: 28372595
[TBL] [Abstract][Full Text] [Related]
13. EU orphan regulation--ten years of application.
Michaux G
Food Drug Law J; 2010; 65(4):639-69, i-ii. PubMed ID: 24479246
[TBL] [Abstract][Full Text] [Related]
14. Inherited retinal dystrophies and orphan designations in the European Union.
Moseley J; Leest T; Larsson K; Magrelli A; Stoyanova-Beninska V
Eur J Ophthalmol; 2024 Mar; ():11206721241236214. PubMed ID: 38500388
[TBL] [Abstract][Full Text] [Related]
15. European regulation on orphan medicinal products: 10 years of experience and future perspectives.
; Westermark K; Holm BB; Söderholm M; Llinares-Garcia J; Rivière F; Aarum S; Butlen-Ducuing F; Tsigkos S; Wilk-Kachlicka A; N'Diamoi C; Borvendég J; Lyons D; Sepodes B; Bloechl-Daum B; Lhoir A; Todorova M; Kkolos I; Kubáčková K; Bosch-Traberg H; Tillmann V; Saano V; Héron E; Elbers R; Siouti M; Eggenhofer J; Salmon P; Clementi M; Krieviņš D; Matulevičiene A; Metz H; Vincenti AC; Voordouw A; Dembowska-Bagińska B; Nunes AC; Saleh FM; Foltánová T; Možina M; Torrent i Farnell J; Beerman B; Mariz S; Evers MP; Greene L; Thorsteinsson S; Gramstad L; Mavris M; Bignami F; Lorence A; Belorgey C
Nat Rev Drug Discov; 2011 May; 10(5):341-9. PubMed ID: 21532564
[TBL] [Abstract][Full Text] [Related]
16. Navigating through orphan medicinal product regulations in EU and US--similarities and differences.
Tiwari J
Regul Toxicol Pharmacol; 2015 Feb; 71(1):63-7. PubMed ID: 25497996
[TBL] [Abstract][Full Text] [Related]
17. Predictors of orphan drug approval in the European Union.
Heemstra HE; de Vrueh RL; van Weely S; Büller HA; Leufkens HG
Eur J Clin Pharmacol; 2008 May; 64(5):545-52. PubMed ID: 18210097
[TBL] [Abstract][Full Text] [Related]
18. Traits, trends and hits of orphan drug designations in cystic fibrosis.
Costa E; Girotti S; van den Ham HA; Cipolli M; van der Ent CK; Taylor-Cousar JL; Leufkens HGM
J Cyst Fibros; 2023 Sep; 22(5):949-957. PubMed ID: 37507282
[TBL] [Abstract][Full Text] [Related]
19. Orphan drug development across Europe: bottlenecks and opportunities.
Heemstra HE; de Vrueh RL; van Weely S; Büller HA; Leufkens HG
Drug Discov Today; 2008 Aug; 13(15-16):670-6. PubMed ID: 18583178
[TBL] [Abstract][Full Text] [Related]
20. Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU.
Fürst-Ladani S; Bührer A; Fürst W; Schober-Ladani N
Handb Exp Pharmacol; 2024; 284():367-387. PubMed ID: 37017789
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
