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Renal and multisystem effectiveness of 3.9 years of migalastat in a global real-world cohort: Results from the followME Fabry Pathfinders registry.
Hughes DA, Sunder-Plassmann G, Jovanovic A, Brand E, West ML, Bichet DG, Pisani A, Nowak A, Torra R, Khan A, Azevedo O, Lehman A, Linhart A, Rutecki J, Giuliano JD, Krusinska E, Nordbeck P. Hughes DA, et al. Among authors: krusinska e. J Inherit Metab Dis. 2025 Jan;48(1):e12771. doi: 10.1002/jimd.12771. Epub 2024 Jul 19. J Inherit Metab Dis. 2025. PMID: 39031114
Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes.
Hughes DA, Bichet DG, Giugliani R, Hopkin RJ, Krusinska E, Nicholls K, Olivotto I, Feldt-Rasmussen U, Sakai N, Skuban N, Sunder-Plassmann G, Torra R, Wilcox WR. Hughes DA, et al. Among authors: krusinska e. J Med Genet. 2023 Jul;60(7):722-731. doi: 10.1136/jmg-2022-108669. Epub 2022 Dec 21. J Med Genet. 2023. PMID: 36543533 Free PMC article.
Corrigendum to "Long-term follow-up of renal function in patients treated with migalastat for Fabry disease" [Bichet et al., MGM Reports; 28 (2021) 100786].
Bichet DG, Torra R, Wallace E, Hughes D, Giugliani R, Skuban N, Krusinska E, Feldt-Rasmussen U, Schiffmann R, Nicholls K. Bichet DG, et al. Among authors: krusinska e. Mol Genet Metab Rep. 2021 Oct 2;29:100807. doi: 10.1016/j.ymgmr.2021.100807. eCollection 2021 Dec. Mol Genet Metab Rep. 2021. PMID: 34934630 Free PMC article.
Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).
Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Paller AS, et al. Among authors: krusinska e. Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3. Orphanet J Rare Dis. 2020. PMID: 32576219 Free PMC article. Clinical Trial.
26 results