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https://www.selleckchem.com/products/heparan-sulfate.html Fabry disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of the lysosomal enzyme α-galactosidase A (α-Gal A) leading to intracellular accumulation of globotriaosylceramide (Gb3). Patients with amenable mutations can be treated with migalastat, a recently approved oral pharmacologic chaperone to increase endogenous α-Gal A activity. We assessed safety along with cardiovascular, renal, and patient-reported outcomes and disease biomarkers in a prospective observational multicenter study after 12 months of migalastat treatment under "real world" conditions. 59 (28 females) patients (34 [57.6%] pre-treated with enzyme replacement therapy) with amenable mutations were recruited. Migalastat was generally safe and well tolerated. Females and males presented with a reduction of left ventricular mass index (primary end point) (-7.2 and -13.7 g/m2 , p=0.0050 and p=0.0061). FD-specific manifestations and symptoms remained stable (all p>0.05). Both sexes presented with a reduction of eGFR (secondary end point) (-6.9 and -5.0 ml/min/1.73 m2 ; p=0.0020 and p=0.0004, respectively), which was most prominent in patients with low blood pressure (p=0.0271). α-Gal A activity increased in male patients by 15% from 29 to 44% of the normal wildtype activity (p=0.0106) and plasma lyso-Gb3 levels were stable in females and males (p=0.3490 and p=0.2009). Re-evaluation of mutations with poor biochemical response revealed no markedly activity increase in a zero activity background. We conclude that therapy with migalastat was generally safe and resulted in an amelioration of left ventricular mass. In terms of impaired renal function, blood pressure control seems to be an unattended important goal. This article is protected by copyright. All rights reserved.KEY POINTS Night time/active phase food restriction for 6 hr impaired glucose intolerance in young male and female mice. Females displayed increase

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