Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease.
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Long-term safety and efficacy of enzyme replacement therapy for Fabry diseaseFabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome SurveyAgalsidase alfa: a review of its use in the management of Fabry diseaseFabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice.A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studiesProduction and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofuscinosisReceptor-mediated endocytosis of α-galactosidase A in human podocytes in Fabry diseaseLong-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cellsReduction of globotriaosylceramide in Fabry disease mice by substrate deprivationDevelopment of a highly sensitive immuno-PCR assay for the measurement of α-galactosidase A protein levels in serum and plasmaMannose 6-phosphate receptor and sortilin mediated endocytosis of α-galactosidase A in kidney endothelial cellsInner ear involvement in Anderson-Fabry disease: long-term follow-up during enzyme replacement therapyIntrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluidStudy of urinary proteomes in Anderson-Fabry disease.Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.Experimental therapies in hypertrophic cardiomyopathyIndividual organelle pH determinations of magnetically enriched endocytic organelles via laser-induced fluorescence detectionThe organizing potential of sphingolipids in intracellular membrane transport.Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Advances in the management of Anderson-Fabry disease: enzyme replacement therapy.Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study.Agalsidase alfa--a preparation for enzyme replacement therapy in Anderson-Fabry disease.Enzyme replacement therapy for Fabry disease: some answers but more questionsPreselective gene therapy for Fabry disease.Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transferUpdate on role of agalsidase alfa in management of Fabry disease.Enzyme therapy for Pompe disease: from science to industrial enterprise.Fabry disease: recent advances in enzyme replacement therapy.Comparative study of structural changes caused by different substitutions at the same residue on α-galactosidase A.Carboxyl-terminal truncations alter the activity of the human α-galactosidase AComparative evaluation of alpha-galactosidase A infusions for treatment of Fabry disease.Enzyme replacement therapy: conception, chaos and culmination.Nano-LC-MS/MS for Quantification of Lyso-Gb3 and Its Analogues Reveals a Useful Biomarker for Fabry Disease.Rapid Immunochromatographic Detection of Serum Anti-α-Galactosidase A Antibodies in Fabry Patients after Enzyme Replacement Therapy.Efficient uptake of recombinant α-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneysCellular and tissue distribution of intravenously administered agalsidase alfa.Innate and Adaptive Immune Response in Fabry DiseaseProduction of recombinant beta-hexosaminidase A, a potential enzyme for replacement therapy for Tay-Sachs and Sandhoff diseases, in the methylotrophic yeast Ogataea minutaWeekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.
P2860
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P2860
Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease.
description
2000 nî lūn-bûn
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2000 թվականի հունվարին հրատարակված գիտական հոդված
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2000年の論文
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2000年論文
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2000年論文
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2000年論文
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2000年论文
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name
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@ast
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@en
type
label
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@ast
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@en
prefLabel
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@ast
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@en
P2093
P2860
P356
P1476
Infusion of alpha-galactosidas ...... n patients with Fabry disease.
@en
P2093
Anderson T
Borowski M
Crutchfield K
Gillespie F
Jeffries NO
P2860
P304
P356
10.1073/PNAS.97.1.365
P407
P577
2000-01-01T00:00:00Z