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Human glyoxylate metabolism revisited: New insights pointing to multi-organ involvement with implications for siRNA-based therapies in primary hyperoxaluria
Wanders, R. J. A., Groothoff, J. W., Deesker, L. J., Salido, E. & Garrelfs, S. F., Jan 2025, In: Journal of inherited metabolic disease. 48, 1, e12817.Research output: Contribution to journal › Review 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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Intrafamilial Disease Heterogeneity in Primary Hyperoxaluria Type 1
Deesker, L. J., Karacoban, H. A., Metry, E. L., Garrelfs, S. F., Bacchetta, J., Boyer, O., Collard, L., Devresse, A., Hayes, W., Hulton, S.-A., Martin-Higueras, C., Moochhala, S. H., Neuhaus, T. J., Oh, J., Prikhodina, L., Sikora, P., Oosterveld, M. J. S., Groothoff, J. W., Mandrile, G. & Beck, B. B., 1 Oct 2024, In: Kidney International Reports. 9, 10, p. 3006-3015 10 p.Research output: Contribution to journal › Article* › Academic › peer-review
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