(Q40317803)

19 September 2017

Limitations of enzyme replacement therapy: current and future (English)

1 reference

enzyme replacement therapy

19 September 2017

0 references

author name string

J E Wraith

series ordinal

1

1 reference

19 September 2017

1 April 2006

1 reference

Journal of Inherited Metabolic Disease

19 September 2017

1 reference

19 September 2017

29

1 reference

19 September 2017

442-447

1 reference

19 September 2017

2-3

1 reference

https://scigraph.springernature.com/pub.10.1007/s10545-006-0239-6

19 September 2017

0 references

cites work

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

1 reference

12 December 2020

NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin

1 reference

12 December 2020

Prediction of neuropathology in mucopolysaccharidosis I patients.

1 reference

12 December 2020

Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey

1 reference

12 December 2020

The natural history of type B Niemann-Pick disease: results from a 10-year longitudinal study

1 reference

12 December 2020

New directions in the treatment of Gaucher disease

1 reference

12 December 2020

Is globotriaosylceramide a useful biomarker in Fabry disease?

1 reference

12 December 2020

Articular chondrocytes from animals with a dermatan sulfate storage disease undergo a high rate of apoptosis and release nitric oxide and inflammatory cytokines: a possible mechanism underlying degenerative joint disease in the mucopolysaccharidoses

1 reference

12 December 2020

Clinical evaluation of chemokine and enzymatic biomarkers of Gaucher disease

1 reference

12 December 2020

The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy.

1 reference

12 December 2020

Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease

1 reference

12 December 2020

Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation

1 reference

12 December 2020

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease

1 reference

12 December 2020

Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts

1 reference

12 December 2020

Gaucher disease: understanding the molecular pathogenesis of sphingolipidoses

1 reference

12 December 2020

Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

1 reference

12 December 2020

Glycosaminoglycan degradation fragments in mucopolysaccharidosis I.

1 reference

12 December 2020

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

1 reference

12 December 2020

Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta

1 reference

12 December 2020

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

1 reference

12 December 2020

Prediction of severity of Gaucher's disease by identification of mutations at DNA level

1 reference

12 December 2020

Keratan sulphate levels in mucopolysaccharidoses and mucolipidoses

1 reference

12 December 2020

Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy.

1 reference

12 December 2020

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

1 reference

12 December 2020

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

1 reference

12 December 2020

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature

1 reference

12 December 2020

Physical performance testing in mucopolysaccharidosis I: a pilot study

1 reference

12 December 2020

Biomarkers in lysosomal storage diseases: a review.

1 reference

12 December 2020

10.1007/S10545-006-0239-6

Identifiers

DOI

1 reference

PubMed ID

16763916

Dimensions Publication ID

19 September 2017

1006818623

0 references

Fatcat ID

release_ehe6zcid4ffxzgzuud623mra54

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/ehe6zcid4ffxzgzuud623mra54

24 November 2022

mapped directly with Wikidata item

PubMed ID

16763916

1 reference

PubMed ID

16763916