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The Effect of Fabry Disease Therapy on Bone Mineral Density.
Aitken T, Tiong MK, Talbot AS, Ruderman I, Nicholls KM. Aitken T, et al. Among authors: nicholls km. Diseases. 2024 May 13;12(5):102. doi: 10.3390/diseases12050102. Diseases. 2024. PMID: 38785757 Free PMC article.
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study.
Linhart A, Dostálová G, Nicholls K, West ML, Tøndel C, Jovanovic A, Giraldo P, Vujkovac B, Geberhiwot T, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Hughes D. Linhart A, et al. Among authors: nicholls k. Orphanet J Rare Dis. 2023 Oct 21;18(1):332. doi: 10.1186/s13023-023-02937-6. Orphanet J Rare Dis. 2023. PMID: 37865771 Free PMC article.
GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals.
Ter Huurne M, Parker BL, Liu NQ, Qian EL, Vivien C, Karavendzas K, Mills RJ, Saville JT, Abu-Bonsrah D, Wise AF, Hudson JE, Talbot AS, Finn PF, Martini PGV, Fuller M, Ricardo SD, Watt KI, Nicholls KM, Porrello ER, Elliott DA. Ter Huurne M, et al. Among authors: nicholls km. Am J Hum Genet. 2023 Sep 7;110(9):1600-1605. doi: 10.1016/j.ajhg.2023.07.013. Epub 2023 Aug 21. Am J Hum Genet. 2023. PMID: 37607539 Free PMC article.
234 results