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Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation
van Kuilenburg, A. B. P., Hollak, C. E. M., Travella, A., Jacobs, M., Gentilini, L. D., Leen, R., der Vlugt, K. M. M. G., Stet, F. S. B., Goorden, S. M. I., van der Veen, S., Criscuolo, M. & Papouchado, M., Jun 2023, In: Drugs in R and D. 23, 2, p. 141-153 13 p.Research output: Contribution to journal › Article › Academic › peer-review
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Repeat expansions nested within tandem CNVs: a unique structural change in GLS exemplifies the diagnostic challenges of non-coding pathogenic variation
Fazal, S., Danzi, M. C., van Kuilenburg, A. B. P., Reich, S., Trasch?tz, A., Bender, B., Leen, R., Toro, C., Usdin, K., Hayward, B., Adams, D. R., van Karnebeek, C. D. M., Ferreira, C. R., D'Sousa, P., Network, U. D., Tekin, M., Z?chner, S. & Synofzik, M., 1 Jan 2023, In: Human Molecular Genetics. 32, 1, p. 46-54 9 p.Research output: Contribution to journal › Article › Academic › peer-review
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Urinary 3-Methoxytyramine Is a Biomarker for MYC Activity in Patients With Neuroblastoma
Verly, I. R. N., Matser, Y. A. H., Leen, R., Meinsma, R., Fiocco, M., Koster, J., Volckmann, R., Savci-Heijink, D., Cangemi, G., Barco, S., Valentijn, L. J., Tytgat, G. A. M. & van Kuilenburg, A. B. P., 2022, In: JCO Precision Oncology. 6, e2000447.Research output: Contribution to journal › Article › Academic › peer-review
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