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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.
Drug Discovery Today|September 1, 2025
Addressing global regulatory challenges in rare disease drug developmentEnrico Costa, Vijayalekshmi Ajith, Ahmed F Al Khaldi, et al.
Gene Therapy|March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designationMaria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in EuropeGloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal ProductsGloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Frontiers in Medicine|September 13, 2021
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's PerspectiveMaria E Sheean, Frauke Naumann-Winter, Giuseppe Capovilla, et al.
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