Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
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Clarifying lysosomal storage diseasesInhibition of endoplasmic reticulum-associated degradation rescues native folding in loss of function protein misfolding diseasesTherapies for the bone in mucopolysaccharidosesImpairment of homeostasis in lysosomal storage disordersProtein replacement therapies for rare diseases: a breeze for regulatory approval?Protein replacement therapy partially corrects the cholesterol-storage phenotype in a mouse model of Niemann-Pick type C2 diseasePleiotropy as the Mechanism for Evolving Novelty: Same Signal, Different ResultHematopoietic stem cell transplantation for Morquio A syndrome.Detection of ligand binding hot spots on protein surfaces via fragment-based methods: application to DJ-1 and glucocerebrosidase.Involvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidosesA selective screening program for the early detection of mucopolysaccharidosis: Results of the FIND project - a 2-year follow-up study.The phytoestrogen genistein modulates lysosomal metabolism and transcription factor EB (TFEB) activationImaging of enzyme replacement therapy using PETTherapies of mucopolysaccharidosis IVA (Morquio A syndrome).Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA.Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Venom Proteins from Parasitoid Wasps and Their Biological Functions.Gene therapy for lysosomal storage diseases (LSDs) in large animal models.Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model.Two-year follow-up of Sanfilippo Disease patients treated with a genistein-rich isoflavone extract: assessment of effects on cognitive functions and general status of patientsA GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations.Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Home-based infusion therapy for patients with Fabry disease.Transplantation of human umbilical mesenchymal stem cells cures the corneal defects of mucopolysaccharidosis VII mice.Fabry disease in children and the effects of enzyme replacement treatment.Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Current therapies for Morquio A syndrome and their clinical outcomes.Genetics and epigenetics of the X chromosome.Genetics and metabolic cardiomyopathies.Genetic biomarkers in hypertrophic cardiomyopathy.Current and potential therapeutic strategies for mucopolysaccharidoses.Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises.Lysosomal storage disorders: A review of the musculoskeletal features.A clinical multicenter study of orofacial features in 26 brazilian patients with different types of mucopolysaccharidosis.Biotherapeutic target or sink: analysis of the macrophage mannose receptor tissue distribution in murine models of lysosomal storage diseases.
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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on January 2007
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@en
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@nl
type
label
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@en
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@nl
prefLabel
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@en
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@nl
P2860
P1433
P1476
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
@en
P2093
Joe T R Clarke
Marianne Rohrbach
P2860
P304
P356
10.2165/00003495-200767180-00005
P577
2007-01-01T00:00:00Z
P6179
1038097749