Amsterdam Gastroenterology Endocrinology Metabolism
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Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries
Rosenberg, N., Post, H. C., Schutte, T., de Visser, S. J., Bartelink, I., Pasmooij, A. M. G., van Laarhoven, H. W. M. & Hollak, C. E. M., 1 Nov 2025, In: ESMO open. 10, 11, 105855.Research output: Contribution to journal › Article › Academic › peer-review
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Application of four pricing models for orphan medicines: a case study for lumasiran
Rosenberg, N., Manders, E., van den Berg, S., Deesker, L. J., Garrelfs, S. F., de Visser, S. J., Groothoff, J. W. & Hollak, C. E. M., 1 Dec 2024, In: Orphanet Journal of Rare Diseases. 19, 1, 485.Research output: Contribution to journal › Article › Academic › peer-review
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Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases
Schoenmakers, D. H., van den Berg, S., Timmers, L., Adang, L. A., Bäumer, T., Bosch, A., van de Casteele, M., Datema, M. R., Dekker, H., Donnelly, C., Driessens, M. H. E., Graessner, H., Greger, V., Haddad, T., Höglinger, G. N. U., van den Hout, H., Jonker, C., Langeveld, M., Lambert, L. J. & Neacy, E. & 12 others, , 22 Aug 2024, In: Neurology. 103, 6, p. 1-12 12 p.Research output: Contribution to journal › Article › Academic › peer-review
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