[HTML][HTML] A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease

S Hwang, BH Lee, WS Kim, DS Kim, CK Cheon… - Medicine, 2022 - journals.lww.com
Background: Fabry disease (FD) is caused by a deficiency in the activity of the lysosomal
enzyme, α-galactosidase A (α-Gal A), which leads to globotriaosylceramide (Gb3) …

Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

R Schiffmann, M Ries, M Timmons… - Nephrology Dialysis …, 2006 - academic.oup.com
Background. Fabry disease is an X-linked disorder of glycosphingolipid catabolism that is
the result of an intracellular deficiency in the lysosomal enzyme α-galactosidase A (α-Gal A) …

[HTML][HTML] Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients

K Tsuboi, H Yamamoto - BMC Pharmacology and Toxicology, 2017 - Springer
Background Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the
α-galactosidase A gene mutations. Enzyme-replacement-therapy (ERT) products for FD …

[HTML][HTML] Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease …

DP Germain, R Giugliani, DA Hughes, A Mehta… - Orphanet journal of rare …, 2012 - Springer
Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the
lysosomal enzyme α-galactosidase A (α-Gal A), which leads to globotriaosylceramide (GL-3) …

Reduction of plasma globotriaosylsphingosine levels after switching from agalsidase alfa to agalsidase beta as enzyme replacement therapy for Fabry disease

O Goker-Alpan, MJ Gambello, GHB Maegawa… - JIMD Reports, Volume …, 2016 - Springer
Introduction: Agalsidase alfa and agalsidase beta, recombinant enzyme preparations for
treatment of Fabry disease (FD), have different approved dosing schedules: 0.2 mg/kg and …

Treatment of Fabry Disease management with migalastat—outcome from a prospective 24 months observational multicenter study (FAMOUS)

M Lenders, P Nordbeck, C Kurschat… - European Heart …, 2022 - academic.oup.com
Aims Fabry disease (FD) is an X-linked lysosomal storage disorder caused by a deficiency
of the lysosomal enzyme α-galactosidase A (GLA/AGAL), resulting in the lysosomal …

Treatment of Fabry's disease with migalastat: outcome from a prospective observational multicenter study (FAMOUS)

M Lenders, P Nordbeck, C Kurschat… - Clinical …, 2020 - Wiley Online Library
Fabry's 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 …

Enzyme replacement therapy for Fabry disease: a systematic review of available evidence

RM Schaefer, A Tylki-Szymańska, MJ Hilz - Drugs, 2009 - Springer
Fabry disease is a progressive and life-threatening glycolipid storage disorder affecting both
males and females. The primary driver of the disease is the accumulation of glycolipids …

[HTML][HTML] Long-term effect of antibodies against infused alpha-galactosidase A in Fabry disease on plasma and urinary (lyso) Gb3 reduction and treatment outcome

SM Rombach, JMFG Aerts, BJHM Poorthuis… - 2012 - journals.plos.org
Introduction Enzyme replacement therapy (ERT) with alpha-Galactosidase A (aGal A) may
cause antibody (AB) formation against aGal A in males with Fabry disease (FD). Anti …

[HTML][HTML] A phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects

R Giugliani, S Waldek, DP Germain, K Nicholls… - Molecular Genetics and …, 2013 - Elsevier
BACKGROUND: Fabry disease (FD) is a genetic disorder resulting from deficiency of the
lysosomal enzyme α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) …