-
Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals
Stolwijk, N. N., Häberle, J., Huidekoper, H. H., Wagenmakers, M. A. E. M., Hollak, C. E. M., Bosch, A. M. & the E-IMD and MetabERN Working Group on the Real-World Use of products for UCD Management, 1 Jan 2025, In: Journal of inherited metabolic disease. 48, 1, e12815.Research output: Contribution to journal › Article › Academic › peer-review
-
Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value-based healthcare approach and systematic benefit-risk assessment
Derks, T. G. J., Venema, A., Köller, C., Bos, E., Overduin, R. J., Stolwijk, N. N., Hofbauer, P., Bolhuis, M. S., van Eenennaam, F., Groen, H., Hollak, C. E. M. & Wortmann, S. B., Mar 2024, In: Journal of inherited metabolic disease. 47, 2, p. 244-254 11 p.Research output: Contribution to journal › Article › Academic › peer-review
-
Food or medicine? A European regulatory perspective on nutritional therapy products to treat inborn errors of metabolism
Stolwijk, N. N., Bosch, A. M., Bouwhuis, N., Häberle, J., van Karnebeek, C., van Spronsen, F. J., Langeveld, M. & Hollak, C. E. M., Nov 2023, In: Journal of inherited metabolic disease. 46, 6, p. 1017-1028 12 p.Research output: Contribution to journal › Article › Academic › peer-review
- All publications
