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2002 3
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221 results

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Page 1
Fabry Disease: Current and Novel Therapeutic Strategies. A Narrative Review.
Palaiodimou L, Kokotis P, Zompola C, Papagiannopoulou G, Bakola E, Papadopoulou M, Zouvelou V, Petras D, Vlachopoulos C, Tsivgoulis G. Palaiodimou L, et al. Curr Neuropharmacol. 2023;21(3):440-456. doi: 10.2174/1570159X20666220601124117. Curr Neuropharmacol. 2023. PMID: 35652398 Free PMC article. Review.
During the last two decades, FD-specific treatments, including two enzyme-replacement-therapies (agalsidase alfa and agalsidase beta) and chaperone treatment with migalastat have been approved for use and allowed for symptoms' stabilization or even disease burden re …
During the last two decades, FD-specific treatments, including two enzyme-replacement-therapies (agalsidase alfa and agalsidase be …
Fabry Disease: The Current Treatment Landscape.
Lenders M, Brand E. Lenders M, et al. Drugs. 2021 Apr;81(6):635-645. doi: 10.1007/s40265-021-01486-1. Epub 2021 Mar 15. Drugs. 2021. PMID: 33721270 Free PMC article. Review.
The lysosomal accumulation of glycosphingolipids, especially globotriaosylceramide (Gb(3)) and globotriaosylsphingosine (lyso-Gb(3), deacylated form), leads to a multisystemic disease with progressive renal failure, cardiomyopathy with potentially malignant cardiac arrhyth …
The lysosomal accumulation of glycosphingolipids, especially globotriaosylceramide (Gb(3)) and globotriaosylsphingosine (lyso-Gb(3), deacyla …
Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study.
Arends M, Biegstraaten M, Wanner C, Sirrs S, Mehta A, Elliott PM, Oder D, Watkinson OT, Bichet DG, Khan A, Iwanochko M, Vaz FM, van Kuilenburg ABP, West ML, Hughes DA, Hollak CEM. Arends M, et al. J Med Genet. 2018 May;55(5):351-358. doi: 10.1136/jmedgenet-2017-104863. Epub 2018 Feb 7. J Med Genet. 2018. PMID: 29437868 Free PMC article.
The risk to develop antibodies was higher for patients treated with agalsidase beta (OR 2.8, P=0.04). LVMI decreased in a higher proportion following the first year of agalsidase beta treatment (OR 2.27, P=0.03), while eGFR slopes were similar. ...A gr …
The risk to develop antibodies was higher for patients treated with agalsidase beta (OR 2.8, P=0.04). LVMI decreased in a high …
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.
Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Banikazemi M, et al. Ann Intern Med. 2007 Jan 16;146(2):77-86. doi: 10.7326/0003-4819-146-2-200701160-00148. Epub 2006 Dec 18. Ann Intern Med. 2007. PMID: 17179052 Free article. Clinical Trial.
BACKGROUND: Fabry disease (alpha-galactosidase A deficiency) is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. OBJECTIVE: To see whether agalsidase beta delays the onset of a compos …
BACKGROUND: Fabry disease (alpha-galactosidase A deficiency) is a rare, X-linked lysosomal storage disorder that can cause early death from …
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Wallace EL, et al. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. J Med Genet. 2024. PMID: 37940383 Free PMC article. Clinical Trial.
BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated glomerular filtration rate (eGFR) slope more negative than -2 mL/min/1.73 m(2)/year who had received agalsidase …
BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease wit …
Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry community.
Wanner C, Ortiz A, Wilcox WR, Hopkin RJ, Johnson J, Ponce E, Ebels JT, Batista JL, Maski M, Politei JM, Martins AM, Banikazemi M, Linhart A, Mauer M, Oliveira JP, Weidemann F, Germain DP. Wanner C, et al. Mol Genet Metab. 2023 Jul;139(3):107603. doi: 10.1016/j.ymgme.2023.107603. Epub 2023 Apr 29. Mol Genet Metab. 2023. PMID: 37236007 Free article. Review.
Leveraging the accumulating evidence base, multidisciplinary collaborations have resulted in the creation of 32 peer-reviewed scientific publications, which have contributed to the greatly expanded knowledge on the onset and progression of FD, its clinical management, the role of …
Leveraging the accumulating evidence base, multidisciplinary collaborations have resulted in the creation of 32 peer-reviewed scientific pub …
Treatment of Fabry Nephropathy: A Literature Review.
Shimohata H, Yamashita M, Yamada K, Hirayama K, Kobayashi M. Shimohata H, et al. Medicina (Kaunas). 2023 Aug 17;59(8):1478. doi: 10.3390/medicina59081478. Medicina (Kaunas). 2023. PMID: 37629768 Free PMC article. Review.
Two treatment options for Fabry nephropathy and cardiopathy are now commercially available: enzyme replacement therapy (agalsidase alpha agalsidase beta, and a biosimilar of agalsidase beta) and pharmacological chaperone therapy (migalastat). In this r …
Two treatment options for Fabry nephropathy and cardiopathy are now commercially available: enzyme replacement therapy (agalsidase alpha …
Treatment in Fabry disease.
López Rodríguez M. López Rodríguez M. Rev Clin Esp (Barc). 2018 Dec;218(9):489-495. doi: 10.1016/j.rce.2018.03.018. Epub 2018 Apr 13. Rev Clin Esp (Barc). 2018. PMID: 29661503 Review. English, Spanish.
Female carriers and males with marginally levels of alpha-galactosidaseA should be treated in case of renal, neurologic o cardiac manifestations. There are two intravenous formulations of human recombinant enzyme, agalsidase alpha and agalsidase beta, showing …
Female carriers and males with marginally levels of alpha-galactosidaseA should be treated in case of renal, neurologic o cardiac man …
Fabry disease.
Thomas AS, Hughes DA. Thomas AS, et al. Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:88-101. Pediatr Endocrinol Rev. 2014. PMID: 25345090 Review.
Clinical features include neuropathic pain and angiokeratoma, with subsequent development of proteinuria, renal failure, left ventricular hypertrophy, arrhythmias and stroke. ...
Clinical features include neuropathic pain and angiokeratoma, with subsequent development of proteinuria, renal failure, left ventric …
The Changing Landscape of Fabry Disease.
Svarstad E, Marti HP. Svarstad E, et al. Clin J Am Soc Nephrol. 2020 Apr 7;15(4):569-576. doi: 10.2215/CJN.09480819. Epub 2020 Mar 4. Clin J Am Soc Nephrol. 2020. PMID: 32132142 Free PMC article. Review. No abstract available.
221 results