PI
Specialization
Inherited Metabolic Diseases
Focus of research
Natural history, pathophysiology and treatment of lysosomal storage disorders
Amsterdam Gastroenterology Endocrinology Metabolism
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Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public–private partnerships
Rosenberg, N., Stolwijk, N. N., van den Berg, S., Heus, J. J., van der Wel, V., van Gelder, T., Bosch, A. M., de Visser, S. J. & Hollak, C. E. M., Sept 2023, In: Journal of inherited metabolic disease. 46, 5, p. 806-816 11 p.Research output: Contribution to journal › Review article › Academic › peer-review
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Access to medicines for rare diseases: A European regulatory roadmap for academia
Rosenberg, N., van den Berg, S., Stolwijk, N. N., Jacobs, B. A. W., Post, H. C., Pasmooij, A. M. G., de Visser, S. J. & Hollak, C. E. M., 2023, In: Frontiers in pharmacology. 14, 1142351.Research output: Contribution to journal › Article › Academic › peer-review
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Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study
Eskes, E. C. B., Beishuizen, C. R. L., Corazolla, E. M., van Middelaar, T., Brands, M. M. M. G., Dekker, H., van de Mheen, E., Langeveld, M., Hollak, C. E. M. & Sjouke, B., 1 Dec 2022, In: Orphanet Journal of Rare Diseases. 17, 1, 383.Research output: Contribution to journal › Article › Academic › peer-review
- All publications
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Clinical and preclinical insights into high-dose ambroxol therapy for Gaucher disease type 2 and 3: A comprehensive systematic review
den Hollander, B., le, H. L., Swart, E. L., Bikker, H., Hollak, C. E. M. & Brands, M. M., 1 Sept 2024, In: Molecular Genetics and Metabolism. 143, 1-2, 108556.Research output: Contribution to journal › Article › Academic › peer-review
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Development, validation and long-term evaluation of a liquid chromatography-tandem mass spectrometry method for simultaneous quantification of amiodarone, desethylamiodarone and mexiletine in human plasma and serum
Braakhuis, M. W. A., Pistorius, M. C. M., Postema, P. G., Hollak, C. E. M. & Swart, E. L., 1 Aug 2024, In: Journal of Chromatography B: Analytical Technologies in the Biomedical and Life Sciences. 1243, 124233.Research output: Contribution to journal › Article › Academic › peer-review
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Illustrating the Financial Consequences of Outcome-Based Payment Models From a Payers Perspective: The Case of Autologous Gene Therapy Atidarsagene Autotemcel (Libmeldy®)
Callenbach, M. H. E., Schoenmakers, D., Vreman, R. A., Vijgen, S., Timmers, L., Hollak, C. E. M., Mantel-Teeuwisse, A. K. & Goettsch, W. G., Aug 2024, In: Value in Health. 27, 8, p. 1046-1057 12 p.Research output: Contribution to journal › Article › Academic › peer-review
- All publications