Projects per year
Personal profile
Research interests
Natural history, pathophysiology and treatment of lysosomal storage disorders
specialisation
Related documents
Collaborations and top research areas from the last five years
Projects
- 1 Finished
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Improving therapeutic efficacy in Fabry patients with anti alphaGAL antibodies
Langeveld, M. (Staff), van Kuilenburg, A. (Staff), Voorberg, J. (Staff), Hollak, C. (Staff) & Van Der Veen, S. S. (Staff)
01/09/2018 → 31/08/2019
Project: Research
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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
Open AccessFile8 Citations (Scopus) -
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
Open Access6 Citations (Scopus) -
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
Open Access2 Citations (Scopus) -
Challenges in the identification and quantification of an unknown impurity in chenodeoxycholic acid drug substance
Bouwhuis, N., Polak, Y., Schimmel, A. M., Bijleveld, Y. A., Giera, M. A., Heijink, M., Vaz, F. M., Bootsma, A. H., Berg-Lammers, L. A. T., Swart, N. E. L., Hollak, C. E. M., Jacobs, B. A. W. & Kemper, E. M., 1 Feb 2025, In: European journal of pharmaceutical sciences. 205, p. 106979 106979.Research output: Contribution to journal › Article › Academic › peer-review
Open Access -
Gene therapy in advanced metachromatic leukodystrophy: tempering expectations
Schoenmakers, D. H., Beerepoot, S., Adang, L. A., Asbreuk, M. A. B. C., Bergner, C. G., Bley, A. E., Boelens, J.-J., Calbi, V., Darling, A., Eklund, E., García Cazorla, Á., Grønborg, S. W., Groeschel, S., van Hasselt, P. M., Hollak, C. E. M., Horgan, C., Jones, S., de Koning, T., Laugwitz, L. & Lindemans, C. & 9 others, , 1 Jan 2025, In: Protein and Cell. 16, 1, p. 12-15 4 p.Research output: Contribution to journal › Article › Academic › peer-review
Open Access
Prizes
Press/Media
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2023: na ontvangst van de KNAW academiepenning
01/01/2023
1 Media contribution
Press/Media: Press / Media
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2022: lancering weesgeneesmiddelentoegangsprotocol
01/01/2022
1 Media contribution
Press/Media: Press / Media
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