[HTML][HTML] The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: a systematic literature review by a European panel of …
Background Enzyme replacement therapy (ERT) with recombinant human α-galactosidase
has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the …
has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the …
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial
M Banikazemi, J Bultas, S Waldek… - Annals of internal …, 2007 - acpjournals.org
Background: Fabry disease (α-galactosidase A deficiency) is a rare, X-linked lysosomal
storage disorder that can cause early death from renal, cardiac, and cerebrovascular …
storage disorder that can cause early death from renal, cardiac, and cerebrovascular …
Natural course of Fabry disease: changing pattern of causes of death in FOS–Fabry Outcome Survey
A Mehta, JTR Clarke, R Giugliani, P Elliott… - Journal of medical …, 2009 - jmg.bmj.com
Background: Fabry disease is a rare X-linked lysosomal storage disorder characterised by
severe multisystemic involvement that leads to major organ failure and premature death in …
severe multisystemic involvement that leads to major organ failure and premature death in …
Effects of enzyme replacement therapy on the cardiomyopathy of Anderson–Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase …
DA Hughes, PM Elliott, J Shah, J Zuckerman… - Heart, 2008 - heart.bmj.com
Background: Anderson–Fabry disease is an X-linked glycosphingolipid storage disorder
caused by deficient activity of the lysosomal enzyme α-galactosidase A. This leads to a …
caused by deficient activity of the lysosomal enzyme α-galactosidase A. This leads to a …
Fabry disease: a review of current management strategies
A Mehta, M Beck, F Eyskens, C Feliciani… - … Journal of Medicine, 2010 - academic.oup.com
Fabry disease is an X-linked inherited condition due to the absence or reduction of α-
galactosidase activity in lysosomes, that results in accumulation of globotriaosylceramide …
galactosidase activity in lysosomes, that results in accumulation of globotriaosylceramide …
Fabry disease
R Schiffmann - Pharmacology & therapeutics, 2009 - Elsevier
Fabry disease, an X-linked disorder of glycosphingolipids that is caused by the deficiency of
α-galactosidase A, is associated with dysfunction of many cell types and includes a systemic …
α-galactosidase A, is associated with dysfunction of many cell types and includes a systemic …
Enzyme replacement therapy for Anderson‐Fabry disease
Background Anderson‐Fabry disease is an X‐linked defect of glycosphingolipid
metabolism. Progressive renal insufficiency is a major source of morbidity, additional …
metabolism. Progressive renal insufficiency is a major source of morbidity, additional …
The role of cardiac imaging in the diagnosis and management of Anderson-Fabry disease
Anderson-Fabry disease (AFD) is a rare X-linked inherited metabolic disorder which results
in a deficiency or absence of the enzyme α-galactosidase A, leading to the accumulation of …
in a deficiency or absence of the enzyme α-galactosidase A, leading to the accumulation of …
[HTML][HTML] Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg
AC Vedder, GE Linthorst, G Houge, JEM Groener… - PloS one, 2007 - journals.plos.org
Background Two different enzyme preparations, agalsidase alfa (ReplagalTM, Shire) and
beta (FabrazymeTM, Genzyme), are registered for treatment of Fabry disease. We compared …
beta (FabrazymeTM, Genzyme), are registered for treatment of Fabry disease. We compared …