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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.
OBJECTIVE: To see whether agalsidase beta delays the onset of a composite clinical outcome of renal, cardiovascular, and cerebrovascular events and death in patients with advanced Fabry disease. ...CONCLUSIONS: Agalsidase-beta
OBJECTIVE: To see whether agalsidase beta delays the onset of a composite clinical outcome of renal, cardiovascular, an …
Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry.
Hopkin RJ, Cabrera G, Charrow J, Lemay R, Martins AM, Mauer M, Ortiz A, Patel MR, Sims K, Waldek S, Warnock DG, Wilcox WR. Hopkin RJ, et al. Mol Genet Metab. 2016 Sep;119(1-2):151-9. doi: 10.1016/j.ymgme.2016.06.007. Epub 2016 Jun 13. Mol Genet Metab. 2016. PMID: 27510433 Free article. Clinical Trial.
METHODS: This longitudinal Fabry Registry study analyzed data from patients with Fabry disease to determine the incidence and type of severe clinical events following initiation of enzyme replacement therapy (ERT) with agalsidase beta
METHODS: This longitudinal Fabry Registry study analyzed data from patients with Fabry disease to determine the inciden …
Pharmacokinetics and pharmacodynamics of JR-051, a biosimilar of agalsidase beta, in healthy adults and patients with Fabry disease: Phase I and II/III clinical studies.
Nakamura K, Kawashima S, Tozawa H, Yamaoka M, Yamamoto T, Tanaka N, Yamamoto R, Okuyama T, Eto Y. Nakamura K, et al. Mol Genet Metab. 2020 Jul;130(3):215-224. doi: 10.1016/j.ymgme.2020.04.003. Epub 2020 May 1. Mol Genet Metab. 2020. PMID: 32389574 Free article. Clinical Trial.
Enzyme replacement therapy is currently the standard therapy for the disease, to which two alpha-galactosidase A formulations have been approved: agalsidase alpha (Replagal, Shire) and agalsidase beta (Fabrazyme, Sanofi). ...In a 52-week, single …
Enzyme replacement therapy is currently the standard therapy for the disease, to which two alpha-galactosidase A formul …
Enzyme replacement therapy stabilized white matter lesion progression in Fabry disease.
Fellgiebel A, Gartenschläger M, Wildberger K, Scheurich A, Desnick RJ, Sims K. Fellgiebel A, et al. Cerebrovasc Dis. 2014;38(6):448-56. doi: 10.1159/000369293. Epub 2014 Dec 11. Cerebrovasc Dis. 2014. PMID: 25502511 Free article. Clinical Trial.
BACKGROUND: The central nervous system manifestations in Fabry disease (FD) include progressive white matter lesions (WMLs) and stroke. ...METHODS: The WML burden and the effect of agalsidase beta 1 mg/kg biweekly on WML progression were assessed longi …
BACKGROUND: The central nervous system manifestations in Fabry disease (FD) include progressive white matter lesions (WMLs) an …
Fabry disease: focus on cardiac manifestations and molecular mechanisms.
Perrot A, Osterziel KJ, Beck M, Dietz R, Kampmann C. Perrot A, et al. Herz. 2002 Nov;27(7):699-702. doi: 10.1007/s00059-002-2429-9. Herz. 2002. PMID: 12439642
Although Fabry disease leads to a complex clinical syndrome, there are studies indicating that manifestations can be limited to the heart. ...ENZYME REPLACEMENT THERAPY: Recent advances in molecular biology and genetic engineering have enabled t …
Although Fabry disease leads to a complex clinical syndrome, there are studies indicating that manifestations can be li …