11 - 20 out of 340Page size: 10
  1. 2022

  2. A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results

    Wasserstein, M., Lachmann, R., Hollak, C., Arash-Kaps, L., Barbato, A., Gallagher, R. C., Giugliani, R., Guelbert, N. B., Ikezoe, T., Lidove, O., Mabe, P., Mengel, E., Scarpa, M., Senates, E., Tchan, M., Villarrubia, J., Chen, Y., Furey, S., Thurberg, B. L., Zaher, A., & 1 othersKumar, M., 1 Jul 2022, In: Genetics in medicine. 24, 7, p. 1425-1436 12 p.

    Research output: Contribution to journal › Article › Academic › peer-review

  3. A vitamin a day keeps the doctor away: The need for high quality pyridoxal-5′-phosphate

    Stolwijk, N. N., Brands, M. M., Smit, L. S., van der Wel, V., Hollak, C. E. M. & van Karnebeek, C. D., 1 Jul 2022, In: European journal of paediatric neurology : EJPN. 39, p. 25-29 5 p.

    Research output: Contribution to journal › Article › Academic › peer-review

  4. Gender- and Age-Associated Differences in Bone Marrow Adipose Tissue and Bone Marrow Fat Unsaturation Throughout the Skeleton, Quantified Using Chemical Shift Encoding-Based Water–Fat MRI

    Beekman, K. M., Regenboog, M., Nederveen, A. J., Bravenboer, N., den Heijer, M., Bisschop, P. H., Hollak, C. E., Akkerman, E. M. & Maas, M., 27 Apr 2022, In: Frontiers in endocrinology. 13, 815835.

    Research output: Contribution to journal › Article › Academic › peer-review

  5. Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression

    van der Veen, S. J., Körver, S., Hirsch, A., Hollak, C. E. M., Wijburg, F. A., Brands, M. M., Tøndel, C., van Kuilenburg, A. B. P. & Langeveld, M., Feb 2022, In: Molecular genetics and metabolism. 135, 2, p. 163-169 7 p.

    Research output: Contribution to journal › Article › Academic › peer-review

  6. Author Reply

    van den Berg, S., de Visser, S. J., Timmers, L. & Hollak, C. E. M., 1 Jan 2022, In: Value in health. 25, 1, p. 158-159 2 p.

    Research output: Contribution to journal › Comment/Letter to the editor › Academic

  7. 2021

  8. Influence of sex and phenotype on cardiac outcomes in patients with Fabry disease

    el Sayed, M., Hirsch, A., Boekholdt, M., van Dussen, L., Datema, M., Hollak, C. & Langeveld, M., 1 Dec 2021, In: Heart. 107, 23, p. 1889-1897 9 p., heartjnl-2020-317922.

    Research output: Contribution to journal › Article › Academic › peer-review

  9. Expanding the clinical spectrum of cerebrotendinous xanthomatosis: Implications for newborn screening, follow-up and treatment

    Hollak, C. E. M., Nov 2021, In: Journal of internal medicine. 290, 5, p. 942-943 2 p.

    Research output: Contribution to journal › Editorial › Academic › peer-review

  10. Drug Repurposing for Rare Diseases: A Role for Academia

    van den Berg, S., de Visser, S., Leufkens, H. G. M. & Hollak, C. E. M., 20 Oct 2021, In: Frontiers in pharmacology. 12, 746987.

    Research output: Contribution to journal › Article › Academic › peer-review

  11. Beoordeling van de prijs van een geneesmiddel. Niet alleen op basis van de waarde voor de patiënten

    Hollak, C. E. M., van den Berg, S., Timmers, L. & Canoy, M., 17 Sep 2021, In: Nederlands tijdschrift voor geneeskunde. 165, 37, D6334.

    Research output: Contribution to journal › Article › Professional

  12. Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia

    van den Berg, S., van der Wel, V., de Visser, S. J., Stunnenberg, B. C., Timmers, L., van der Ree, M. H., Postema, P. G. & Hollak, C. E. M., Jul 2021, In: Value in health. 24, 7, p. 925-929 5 p.

    Research output: Contribution to journal › Article › Academic › peer-review

Previous 1 2 3 4 5 6 7 8 ...34 Next

ID: 61215