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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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Perspectives and Update on the Global Shortage of Verteporfin (Visudyne®): Ophthalmology and Therapy
Sirks, M. J., Subhi, Y., Rosenberg, N., Hollak, C. E. M., Boon, C. J. F., Diederen, R. M. H., Yzer, S., Ossewaarde-van Norel, J., de Jong-Hesse, Y., Schlingemann, R. O., Moss, R. J. & van Dijk, E. H. C., 2024, In: Ophthalmol. Ther.. 13, 7, p. 1821-1831 11 p.Research output: Contribution to journal › Comment/Letter to the editor* › Academic
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