Showing results (11-20 of 31) with videos related to

Sort By:
Pageof 4
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.
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.
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.
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.
Frontiers in Medicine|July 1, 2022
A Comparison of Post-marketing Measures Imposed by Regulatory Agencies to Confirm the Tissue-Agnostic ApproachJorn Mulder, Odile C van Stuijvenberg, Paula B van Hennik, et al.
Drug Discovery Today|October 13, 2017
Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in EuropeLaura Fregonese, Lesley Greene, Matthias Hofer, et al.
Nucleic Acid Therapeutics|August 11, 2017
Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding IssuesAnnemieke Aartsma-Rus, Volker Straub, Robert Hemmings, et al.
Clinical Pharmacology and Therapeutics|June 20, 2020
Can a Multistakeholder Prioritization Structure Support Regulatory Decision Making? A Review of Pediatric Oncology Strategy Forums Reflecting on Challenges and Opportunities of this ConceptDominik Karres, Giovanni Lesa, Franca Ligas, et al.
Drug Discovery Today|November 9, 2025
Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approvalAnne R Pariser, Violeta Stoyanova-Beninska, Oxana Iliach, et al.
Pageof 4