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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PHYOX3: Nedosiran Long-Term Safety and Efficacy in Patients With Primary Hyperoxaluria Type 1 by John C. Lieske, Gema Ariceta, Jaap W. Groothoff, Graham Lipkin, Shabbir H. Moochhala, Gesa Schalk, Anne-Laure Sellier-Leclerc, Sara Estupiñan Torres, Verity Rawson, Jing Zhou, Bernd Hoppe

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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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Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial by Jeffrey M. Saland, John C. Lieske, Jaap W. Groothoff, Yaacov Frishberg, Hadas Shasha-Lavsky, Daniella Magen, Shabbir H. Moochhala, Eva Simkova, Martin Coenen, Wesley Hayes, Julien Hogan, Anne-Laure Sellier-Leclerc, Richard Willey, John M. Gansner, Sally-Anne Hulton

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Opportunities in Primary and Enteric Hyperoxaluria at the Cross-Roads Between the Clinic and Laboratory by Barbara Cellini, Michelle A. Baum, Yaacov Frishberg, Jaap W. Groothoff, Peter C. Harris, Sally A. Hulton, Felix Knauf, John Knight, John C. Lieske, W. Todd Lowther, Shabbir Moochhala, Lama Nazzal, Gregory E. Tasian, Jonathan M. Whittamore, David J. Sas

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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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