Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
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Inhibition of endoplasmic reticulum-associated degradation rescues native folding in loss of function protein misfolding diseasesProtein replacement therapy partially corrects the cholesterol-storage phenotype in a mouse model of Niemann-Pick type C2 diseaseMultiplex newborn screening for Pompe, Fabry, Hunter, Gaucher, and Hurler diseases using a digital microfluidic platform.Current and emerging treatments and surgical interventions for Morquio A syndrome: a reviewEnhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expressionBiomarker analysis of Morquio syndrome: identification of disease state and drug responsive markersActivities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.Rapid assays for Gaucher and Hurler diseases in dried blood spots using digital microfluidics.Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.
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P2860
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on August 2009
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@en
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@nl
type
label
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@en
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@nl
prefLabel
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@en
Current enzyme replacement therapy for the treatment of lysosomal storage diseases.
@nl
P1433
P1476
Current enzyme replacement therapy for the treatment of lysosomal storage diseases
@en
P2093
David F Kronn
Elizabeth R Lim-Melia
P304
P356
10.3928/00904481-20090723-09
P577
2009-08-01T00:00:00Z