Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain. - Wikidata - wikimedia - TriplyDB

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.

http://www.wikidata.org/entity/Q36767452

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Enzyme replacement therapy for Anderson-Fabry disease Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry.[Globosides as key players in the pathophysiology of Shiga toxin-associated acute kidney failure and Fabry disease].Fabry nephropathy: a review - how can we optimize the management of Fabry nephropathy?Effectiveness of enzyme replacement therapy in Fabry disease: Long term experience in Argentina.Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.Depletion of globosides and isoglobosides fully reverts the morphologic phenotype of Fabry disease Fabry disease: multidisciplinary evaluation after 10 years of treatment with agalsidase Beta.Prevalence of Raynaud phenomenon and nailfold capillaroscopic abnormalities in Fabry disease: a cross-sectional study Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document.Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa.Innate and Adaptive Immune Response in Fabry Disease Quality of life in patients with Fabry disease: a systematic review of the literature.The Large Phenotypic Spectrum of Fabry Disease Requires Graduated Diagnosis and Personalized Therapy: A Meta-Analysis Can Help to Differentiate Missense Mutations Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies.Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis Clinical evidence for orphan medicinal products-a cause for concern?Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease.Fabry disease: a rare cause of neuropathic pain.Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis: effectiveness of ERT in different disease stages.Unravelling the mechanism of action of enzyme replacement therapy in Fabry disease.Clinical characteristics and mutation spectrum of GLA in Korean patients with Fabry disease by a nationwide survey: Underdiagnosis of late-onset phenotype.Fabry Disease: Recognition, Diagnosis, and Treatment of Neurological Features.Characterization of Classical and Nonclassical Fabry Disease: A Multicenter Study.Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.GLA mutation as a risk factor for later life small vessel ischaemic disease.Screening of Fabry disease in patients with end-stage renal disease of unknown etiology: the first Thailand study.The value of ECG parameters as markers of treatment response in Fabry cardiomyopathy.Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study.E-Learning for Rare Diseases: An Example Using Fabry Disease.Phenotype, disease severity and pain are major determinants of quality of life in Fabry disease: results from a large multicenter cohort study.Echocardiographic and clinical findings in patients with Fabry disease during long-term enzyme replacement therapy: a nationwide Danish cohort study.Pathomechanisms of renal Fabry disease.Enzymes as Immunotherapeutics.Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study.Hearing loss in adult patients with Fabry disease treated with enzyme replacement therapy.Long-term enzyme replacement therapy for Fabry disease: efficacy and unmet needs in cardiac and renal outcomes.Fabry disease: is there a role for enzyme replacement therapy?

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P2860

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.

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Bouwien E Smid

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Carla E M Hollak

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Gabor E Linthorst

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Machtelt G Bouwman

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P2860

Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg

New equations to estimate GFR in children with CKD

A more accurate method to estimate glomerular filtration rate from serum creatinine: a new prediction equation. Modification of Diet in Renal Disease Study Group

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

The clinical importance of white matter hyperintensities on brain magnetic resonance imaging: systematic review and meta-analysis

Cardiovascular events in patients with fabry disease natural history data from the fabry registry.

Response of women with Fabry disease to enzyme replacement therapy: comparison with men, using data from FOS--the Fabry Outcome Survey.

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

Fabry's disease: alpha-galactosidase deficiency.

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

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Enzyme replacement therapy

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