New opportunities for accessing promising non-oncological orphan drugs.
作者信息
Hollak Carla E M, Reparon-Schuijt Carelle C, Verdeyen Kathelijn, Deesker Lisa J, Groothoff Jaap W, Vos Josephine M I, Huisman Atse H, van der Flier Silvia, Pisters-van Roy Anke A M G
机构信息
Department of Endocrinology and Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, the Netherlands.
Medicines for Society, Platform at Amsterdam UMC Location University of Amsterdam, Amsterdam, the Netherlands.
出版信息
Lancet Reg Health Eur. 2025 Mar 21;52:101275. doi: 10.1016/j.lanepe.2025.101275. eCollection 2025 May.
Abstract
摘要
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