Application of four pricing models for orphan medicines: a case study for lumasiran by Noa Rosenberg, Evert Manders, Sibren van den Berg, Lisa J. Deesker, Sander F. Garrelfs, Saco J. de Visser, Jaap W. Groothoff, Carla E. M. Hollak

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New opportunities for accessing promising non-oncological orphan drugs by Carla E.M. Hollak, Carelle C. Reparon-Schuijt, Kathelijn Verdeyen, Lisa J. Deesker, Jaap W. Groothoff, Josephine M.I. Vos, Atse H. Huisman, Silvia van der Flier, Anke A.M.G. Pisters-van Roy

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Intrafamilial Disease Heterogeneity in Primary Hyperoxaluria Type 1 by Lisa J. Deesker, Hazal A. Karacoban, Elisabeth L. Metry, Sander F. Garrelfs, Justine Bacchetta, Olivia Boyer, Laure Collard, Arnaud Devresse, Wesley Hayes, Sally-Anne Hulton, Cristina Martin-Higueras, Shabbir H. Moochhala, Thomas J. Neuhaus, Jun Oh, Larisa Prikhodina, Przemyslaw Sikora, Michiel J.S. Oosterveld, Jaap W. Groothoff, Giorgia Mandrile, Bodo B. Beck

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