Current and potential therapeutic strategies for mucopolysaccharidoses.
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Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersDose responsive effects of subcutaneous pentosan polysulfate injection in mucopolysaccharidosis type VI rats and comparison to oral treatmentExtraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.Mesenchymal Stem Cells Yield Transient Improvements in Motor Function in an Infant Rhesus Macaque with Severe Early-Onset Krabbe Disease.Genetic rescue of an endangered domestic animal through outcrossing with closely related breeds: A case study of the Norwegian LundehundSpinal involvement in mucopolysaccharidoses: a review.Assessment and diagnosis of suspected glaucoma in patients with mucopolysaccharidosis.Emerging drugs for the treatment of mucopolysaccharidoses.Subcutaneous implantation of microencapsulated cells overexpressing α-L-iduronidase for mucopolysaccharidosis type I treatment.Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS).Anesthetic care and perioperative complications in children with Sanfilipo Syndrome Type A.Transduction Profile of the Marmoset Central Nervous System Using Adeno-Associated Virus Serotype 9 Vectors.Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis.Development of a fluorometric microtiter plate-based enzyme assay for arylsulfatase B (MPS VI) using dried blood spots.Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders.Presentation and Treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome).Neural cells generated from human induced pluripotent stem cells as a model of CNS involvement in mucopolysaccharidosis type II.Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe.Non-sibling hematopoietic stem cell transplantation using myeloablative conditioning regimen in children with Maroteaux-Lamy syndrome: A brief report.Cerebral magnetic resonance findings during enzyme replacement therapy in mucopolysaccharidosis.Cyclodextrin encapsulation of daidzein and genistein by grinding: implication on the glycosaminoglycan accumulation in mucopolysaccharidosis type II and III fibroblasts.Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses.
P2860
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P2860
Current and potential therapeutic strategies for mucopolysaccharidoses.
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article científic
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article scientifique
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articol științific
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articolo scientifico
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artigo científico
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artigo científico
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artigo científico
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artikel ilmiah
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artículo científico
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name
Current and potential therapeutic strategies for mucopolysaccharidoses.
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type
label
Current and potential therapeutic strategies for mucopolysaccharidoses.
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prefLabel
Current and potential therapeutic strategies for mucopolysaccharidoses.
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P2860
P356
P1476
Current and potential therapeutic strategies for mucopolysaccharidoses.
@en
P2860
P304
P356
10.1111/JCPT.12136
P577
2014-02-25T00:00:00Z