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Journal of the American Society of Nephrology, ISSN 1046-6673, 12/2018, Volume 29, Issue 12, pp. 2879 - 2889
Background Use of enzyme replacement therapy (ERT) to treat Fabry disease, caused by deficient lysosomal alpha-galactosidase A activity, can lead to formation... 
IMMUNE-RESPONSE | EFFICACY | SAFETY | HUMAN ALPHA-GALACTOSIDASE | UROLOGY & NEPHROLOGY | AGALSIDASE-ALPHA | SERUM | SUBSTRATE REDUCTION THERAPY | BETA
Journal Article
Journal Article
LANCET, ISSN 0140-6736, 05/2010, Volume 375, Issue 9725, pp. 1523 - 1524
Journal Article
Drug Discovery Today, ISSN 1359-6446, 06/2018, Volume 23, Issue 6, pp. 1251 - 1257
Fabry disease (FD) is a rare X-chromosome-linked lysosomal storage disorder. Although initial expectations of enzyme replacement therapy (ERT) were high, it is... 
ENZYME-REPLACEMENT THERAPY | EFFICACY | PEDIATRIC-PATIENTS | CONSENSUS | UNCERTAIN DIAGNOSIS | FOLLOW-UP | PHARMACOLOGY & PHARMACY | AGALSIDASE-ALPHA | OPEN-LABEL | MANIFESTATIONS | BETA | Enzyme Replacement Therapy | alpha-Galactosidase - therapeutic use | Fabry Disease - drug therapy | Isoenzymes - therapeutic use | Humans | Medical colleges | Enzymes
Journal Article
CLINICA CHIMICA ACTA, ISSN 0009-8981, 12/2010, Volume 411, Issue 23-24, pp. 1906 - 1914
Background: Fabry disease is characterized by accumulation of glycosphingolipids, such as globotriaosylceramide (Gb(3)), in many tissues and body fluids. A... 
Lyso-Gb | SAFETY | Fabry disease | PHENOTYPE | Liquid chromatography-tandem mass spectrometry | PREVALENCE | LYSOSOMAL STORAGE DISORDERS | TANDEM MASS-SPECTROMETRY | Time-of-flight mass spectrometry | NEPHROPATHY | LC-MS/MS | GLOMERULAR-FILTRATION-RATE | AGALSIDASE-ALPHA | MEDICAL LABORATORY TECHNOLOGY | ENZYME REPLACEMENT THERAPY | ALPHA-GALACTOSIDASE
Journal Article
Molecular Therapy, ISSN 1525-0016, 04/2019, Volume 27, Issue 4, pp. 878 - 889
Fabry disease is a lysosomal storage disorder caused by the deficiency of α-galactosidase A. Enzyme deficiency results in a progressive decline in renal and... 
delivery | lipid nanoparticle | mRNA | enzyme replacement therapy | Fabry | MEDICINE, RESEARCH & EXPERIMENTAL | GLYCOSYLATION | BETA | NANOPARTICLES | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | GENETICS & HEREDITY | AGALSIDASE-ALPHA | MICE | OPTIMIZATION | EXPRESSION | ALPHA-GALACTOSIDASE | TARGETED DELIVERY
Journal Article
Journal of the American Society of Echocardiography, ISSN 0894-7317, 06/2018, Volume 31, Issue 6, pp. 639 - 649.e2
Journal Article
American Journal of Kidney Diseases, ISSN 0272-6386, 2008, Volume 51, Issue 5, pp. 767 - 776
Journal Article
European journal of neurology : the official journal of the European Federation of Neurological Societies, ISSN 1351-5101, 1/2014, Volume 21, Issue 1, pp. 3 - 4
Journal Article
Genetics in Medicine, ISSN 1098-3600, 09/2006, Volume 8, Issue 9, pp. 539 - 548
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2016, Volume 117, Issue 2, pp. 66 - 83
Journal Article