Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. - Wikidata - awgldk - TriplyDB

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.

http://www.wikidata.org/entity/Q35550696

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Embolic Strokes of Unknown Source and Cryptogenic Stroke: Implications in Clinical Practice Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry.Correlations between Endomyocardial Biopsies and Cardiac Manifestations in Taiwanese Patients with the Chinese Hotspot IVS4+919G>A Mutation: Data from the Fabry Outcome Survey.Effectiveness of enzyme replacement therapy in Fabry disease: Long term experience in Argentina.Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients.Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.Case report of a 45-year old female Fabry disease patient carrying two alpha-galactosidase A gene mutation alleles.Uric Acid as a Marker of Mortality and Morbidity in Fabry Disease.Urinary Podocyte Loss Is Increased in Patients with Fabry Disease and Correlates with Clinical Severity of Fabry Nephropathy Antiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy.Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up.Novel GLA Deletion in a Cypriot Female Presenting with Cornea Verticillata Pain in Fabry Disease: Practical Recommendations for Diagnosis and Treatment.Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease.Clinical characteristics and mutation spectrum of GLA in Korean patients with Fabry disease by a nationwide survey: Underdiagnosis of late-onset phenotype.Causally treatable, hereditary neuropathies in Fabry's disease, transthyretin-related familial amyloidosis, and Pompe's disease.The Impact of Fabry Disease on Reproductive Fitness.Newborn Screening for Lysosomal Storage Disorders: Views of Genetic Healthcare Providers.Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease.Therapeutic Strategies Targeting Inherited Cardiomyopathies.Parapelvic cysts, a distinguishing feature of renal Fabry disease.Screening Fabry's disease in chronic kidney disease patients not on dialysis: a multicenter study.Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors Biomarkers and Imaging Findings of Anderson-Fabry Disease-What We Know Now.A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events.Fabry disease: characterisation of the plasma proteome pre- and post-enzyme replacement therapy.Prominent regression of corneal deposits in Fabry disease 16 years after initiation of enzyme replacement therapy.Long-Term Dose-Dependent Agalsidase Effects on Kidney Histology in Fabry Disease.Ten-year-long enzyme replacement therapy shows a poor effect in alleviating giant leg ulcers in a male with Fabry disease.Podocyturia is significantly elevated in untreated vs treated Fabry adult patients.Significant improvement in Fabry disease podocytopathy after 3 years of treatment with agalsidase beta.[Anterior segment findings with far-reaching consequences].Surges in proteinuria are associated with plasma GL-3 elevations in a young patient with classic Fabry disease.Variable phenotypic presentations of renal involvement in Fabry disease: a case series.Association and diagnostic utility of diastolic dysfunction and myocardial fibrosis in patients with Fabry disease Fabry disease in a Japanese population-molecular and biochemical characteristics

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Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.

http://www.wikidata.org/entity/Q35550696

description

2015 nî lūn-bûn

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2015 թուականի Մարտին հրատարակուած գիտական յօդուած

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2015 թվականի մարտին հրատարակված գիտական հոդված

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2015年の論文

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2015年論文

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2015年論文

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2015年論文

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2015年論文

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2015年論文

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2015年论文

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Ten-year outcome of enzyme rep ...... n patients with Fabry disease.

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JMEDGENET-2014-102797

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Journal of Medical Genetics

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Ten-year outcome of enzyme rep ...... n patients with Fabry disease.

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C Ronald Scott

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David G Warnock

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Gabor E Linthorst

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Joel Charrow

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Judy Kempf

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Nathalie Guffon

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Neal J Weinreb

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Robert J Desnick

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Roberta Lemay

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Seymour Packman

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P2860

Long-term safety and efficacy of enzyme replacement therapy for Fabry disease

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry.

Stroke in Fabry disease frequently occurs before diagnosis and in the absence of other clinical events: natural history data from the Fabry Registry.

End-stage renal disease in patients with Fabry disease: natural history data from the Fabry Registry.

Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data.

Enzyme replacement therapy in Fabry disease: a randomized controlled trial.

Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.

Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications

Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males

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353-358

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10.1136/JMEDGENET-2014-102797

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5

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Dominique P Germain

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2015-03-20T00:00:00Z

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273832788

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25795794

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Enzyme replacement therapy

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