Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties.

Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties.

Item
http://www.wikidata.org/entity/Q34281022
Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience.Laronidase replacement therapy and left ventricular function in mucopolysaccharidosis I.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Gene therapy for lysosomal storage diseases (LSDs) in large animal models.Glycemic control and chronic dosing of rhesus monkeys with a fusion protein of iduronidase and a monoclonal antibody against the human insulin receptor.IgG-enzyme fusion protein: pharmacokinetics and anti-drug antibody response in rhesus monkeys.Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys.Mannose 6-phosphate receptor-mediated uptake is defective in acid sphingomyelinase-deficient macrophages: implications for Niemann-Pick disease enzyme replacement therapy.Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model.alpha-L-iduronidase therapy for mucopolysaccharidosis type I.Two-dimensional NMR spectroscopy of urinary glycosaminoglycans from patients with different mucopolysaccharidoses.Sleep disturbance in Sanfilippo syndrome: a parental questionnaire study.Selective plasma pharmacokinetics and brain uptake in the mouse of enzyme fusion proteins derived from species-specific receptor-targeted antibodies.Characterization of a Case of Pigmentary Retinopathy in Sanfilippo Syndrome Type IIIA Associated with Compound Heterozygous Mutations in the SGSH Gene.Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
P2860

Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties.

Item
http://www.wikidata.org/entity/Q34281022
description
2001 nî lūn-bûn
@nan
2001 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2001年の論文
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2001年論文
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2001年論文
@zh-hant
2001年論文
@zh-hk
2001年論文
@zh-mo
2001年論文
@zh-tw
2001年论文
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name
Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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type
label
Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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P1476
P2093
P2888
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P6179
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Enzyme replacement therapy in ...... ess and emerging difficulties.
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P2093
P2888
P304
P31
P356
P433
P478
P577
P6179
P698
P921