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Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression.
van der Veen SJ, Körver S, Hirsch A, Hollak CEM, Wijburg FA, Brands MM, Tøndel C, van Kuilenburg ABP, Langeveld M. van der Veen SJ, et al. Among authors: brands mm. Mol Genet Metab. 2022 Feb;135(2):163-169. doi: 10.1016/j.ymgme.2021.12.004. Epub 2021 Dec 17. Mol Genet Metab. 2022. PMID: 35033446 Free article.
Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials.
Müller AR, den Hollander B, van de Ven PM, Roes KCB, Geertjens L, Bruining H, van Karnebeek CDM, Jansen FE, de Wit MCY, Ten Hoopen LW, Rietman AB, Dierckx B, Wijburg FA, Boot E, Brands MMG, van Eeghen AM. Müller AR, et al. BMC Psychiatry. 2024 Jan 4;24(1):23. doi: 10.1186/s12888-023-05422-3. BMC Psychiatry. 2024. PMID: 38177999 Free PMC article.
31 results