Showing results (1-10 of 26) with videos related to

Sort By:
Pageof 3
European Journal of Ophthalmology|March 19, 2024
Inherited retinal dystrophies and orphan designations in the European UnionJane Moseley, Tim Leest, Kristina Larsson, et al.
Frontiers in Medicine|July 12, 2021
Regulatory Standards in Orphan Medicinal Product Designation in the EUStelios Tsigkos, Segundo Mariz, Maria Elzbieta Sheean, et al.
British Journal of Clinical Pharmacology|March 27, 2021
Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspectiveFrancesca Tomeo, Segundo Mariz, Angelo Loris Brunetta, et al.
Frontiers in Medicine|June 1, 2017
Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in EuropeAndreas M Farkas, Segundo Mariz, Violeta Stoyanova-Beninska, et al.
Journal of Clinical Pharmacology|September 8, 2018
Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective AnalysisYvonne Schuller, Christine Gispen-de Wied, Carla E M Hollak, et al.
European Journal of Pharmaceutical Sciences : Official Journal of the European Federation for Pharmaceutical Sciences|August 7, 2024
Feasibility of disease terminology systems for mapping orphan conditions and therapeutic indications of designated orphan medicines in the European UnionKeerti D Jadoenathmisier, Violeta Stoyanova-Beninska, Inge M Soons, et al.
Frontiers in Oncology|January 6, 2022
Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan DesignationJorn Mulder, Tobias van Rossum, Segundo Mariz, et al.
Orphanet Journal of Rare Diseases|November 30, 2018
Oncologic orphan drugs approved in the EU - do clinical trial data correspond with real-world effectiveness?Yvonne Schuller, Marieke Biegstraaten, Carla E M Hollak, et al.
Drug Discovery Today|February 21, 2026
Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines AgencyNoa Rosenberg, Carla E M Hollak, V A Haverhoek, et al.
Journal of Neuromuscular Diseases|November 18, 2025
Paediatric medicines development for Duchenne muscular dystrophy: An EU regulatory perspectiveSylvie Benchetrit, Céline Chu, Dimitrios Athanasiou, et al.
Pageof 3