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Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the

Noa Rosenberg1, Carla E M Hollak2, V A Haverhoek3

  • 1Medicines for Society (Medicijn voor de Maatschappij), Platform at Amsterdam UMC - University of Amsterdam, Amsterdam, The Netherlands; Department of Endocrinology and Metabolism, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, MetabERN, University of Amsterdam, Amsterdam, The Netherlands; Dutch Medicines Evaluation Board, Utrecht, The Netherlands.

Drug Discovery Today
|February 21, 2026
PubMed

Keywords:
medicines developmentorphan designationorphan medicineprotocol assistancerare diseaseregulatory interaction

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