Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain (Q36767452)

16 August 2017

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

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enzyme replacement therapy

16 August 2017

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Saskia M Rombach

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16 August 2017

Bouwien E Smid

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16 August 2017

Machtelt G Bouwman

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16 August 2017

Gabor E Linthorst

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16 August 2017

Marcel G W Dijkgraaf

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16 August 2017

Carla E M Hollak

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16 August 2017

25 March 2013

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Orphanet Journal of Rare Diseases

16 August 2017

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16 August 2017

8

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16 August 2017

47

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https://scigraph.springernature.com/pub.10.1186/1750-1172-8-47

16 August 2017

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cites work

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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The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.

29 September 2017

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Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

29 September 2017

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

29 September 2017

1 reference

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Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

29 September 2017

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

29 September 2017

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Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT).

29 September 2017

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

29 September 2017

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

29 September 2017

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

29 September 2017

1 reference

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New equations to estimate GFR in children with CKD

29 September 2017

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Elevated globotriaosylsphingosine is a hallmark of Fabry disease.

29 September 2017

1 reference

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

29 September 2017

1 reference

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

29 September 2017

1 reference

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National Kidney Foundation practice guidelines for chronic kidney disease: evaluation, classification, and stratification

29 September 2017

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Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

29 September 2017

1 reference

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

29 September 2017

1 reference

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Normal values of M mode echocardiographic measurements of more than 2000 healthy infants and children in central Europe

29 September 2017

1 reference

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

29 September 2017

1 reference

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Fabry's Disease: Alpha-Galactosidase Deficiency

29 September 2017

1 reference

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Left Ventricular Mass as a Measure of Preclinical Hypertensive Disease

29 September 2017

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Baseline characteristics of patients enrolled in the Canadian Fabry Disease Initiative.

29 September 2017

1 reference

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Heart function in patients with chronic glomerulonephritis and mildly to moderately impaired renal function. An echocardiographic study.

27 June 2018

1 reference

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Echocardiographic assessment of left ventricular hypertrophy: comparison to necropsy findings.

27 June 2018

1 reference

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Inappropriately high left ventricular mass in patients with type 2 diabetes mellitus and no overt cardiac disease. The DYDA study.

27 June 2018

1 reference

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Plasma globotriaosylsphingosine: diagnostic value and relation to clinical manifestations of Fabry disease

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3626869

Age- and gender-specific reference values of estimated GFR in Caucasians: the Nijmegen Biomedical Study.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3626869

Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3626869

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females

5 September 2018

1 reference

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Quantification of globotriaosylsphingosine in plasma and urine of fabry patients by stable isotope ultraperformance liquid chromatography-tandem mass spectrometry

5 September 2018

1 reference

12 December 2020

Enzymatic Defect in Fabry's Disease

1 reference

12 December 2020

An atypical variant of Fabry's disease in men with left ventricular hypertrophy

1 reference

12 December 2020

The variation of morphological and functional cardiac manifestation in Fabry disease: potential implications for the time course of the disease

1 reference

12 December 2020

Impact of enzyme replacement therapy on cardiac morphology and function and late enhancement in Fabry's cardiomyopathy

1 reference

12 December 2020

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

1 reference

12 December 2020

Long-term effects of enzyme replacement therapy on fabry cardiomyopathy: evidence for a better outcome with early treatment

1 reference

12 December 2020

Identifiers

DOI

10.1186/1750-1172-8-47

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Dimensions Publication ID

16 August 2017

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16 August 2017

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