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Stroke and Transient Ischemic Attacks of the Brain and Eye
Published in Philip B. Gorelick, Fernando D. Testai, Graeme J. Hankey, Joanna M. Wardlaw, Hankey's Clinical Neurology, 2020
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by GLA gene mutations that result in α-galactosidase deficiency and subsequent accumulation of globotriaosylceramide in various organs (Table 12.23). Progressive accumulation of globotriaosylceramide in endothelial and vascular smooth muscle cells causes progressive stenosis and occlusion of small arterial vessels. Furthermore, the large vessels dilate, resulting in dolichoectatic changes and flow stagnation, particularly in the posterior circulation (e.g. basilar artery), increasing the risk of artery-to-artery embolism and vessel thrombosis. Hence, FD is associated with both cerebral macroangiopathy and microangiopathy, which leads to an increased incidence of stroke and first stroke at a younger age than the general population. Preliminary studies suggest that enzyme replacement therapy (e.g. agalsidase alfa or agalsidase beta) may reduce the risk of stroke.8
Biocatalytic Nanoreactors for Medical Purposes
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2019
Oscar González-Davis, Chauhan Kanchan, Rafael Vazquez-Duhalt
Fabry disease is a disorder linked to glycosphingolipid metabolism caused by α-galactosidase A (α-Gal) deficiency. The successful use of ERT for Fabry disease has been reported (Alfadhel and Sirrs, 2011). Recombinant agalsidase alfa and agalsidase beta have been compared for Fabry disease treatment (Arends et al., 2018). The authors found no difference between patients treated with either agalsidase, and a more pronounced reduction of storage materials was found with agalsidase beta.
Long-term safety and efficacy of agalsidase beta in Japanese patients with Fabry disease: aggregate data from two post-authorization safety studies
Published in Expert Opinion on Drug Safety, 2021
Mina Tsurumi, Shinya Suzuki, Jiro Hokugo, Kazuo Ueda
The Special Drug Use Investigation of agalsidase beta analyzed the safety and efficacy of long-term agalsidase beta use in Japanese patients with Fabry disease. The Drug Use Investigation of agalsidase beta was conducted using an all-case surveillance method and analyzed the safety profile in all Japanese patients with Fabry disease who were not included in the Special Drug Use Investigation of agalsidase beta.
Visual outcome, ocular findings, and visual quality of life in patients with Fabry disease
Published in Ophthalmic Genetics, 2022
Mattias Nilsson, Hani Tavakoli Kolagari, David Epstein, Branka Samolov, Monica Olsson, Karin Naess, Mikael Oscarson, Kristina Teaer Fahnehjelm
A majority of patients fulfilled the criteria for Fabry-specific treatment. Among males of all ages, 11 were treated with agalsidase beta, one with agalsidase alpha, and one with the pharmacologic chaperone migalastat. Among the females, four patients were treated with agalsidase beta, one with agalsidase alpha, and one migalastat (Table 1).