Substrate reduction therapy

Substrate reduction therapy

Näytä muut versiot (1)

The glycosphingolipidoses are a family of storage diseases that arise due to incomplete catabolism of glycosphingolipids (GSLs) in the lysosome (Wraith, 2002). The majority are autosomal recessive disorders and result from mutations in the genes that encode the catabolic enzymes of the lysosome (Win...

Bibliografiset tiedot
Päätekijät:	Platt, F, Butters, T
Aineistotyyppi:	Book section
Julkaistu:	Springer US 2007

Samankaltaisia teoksia

Substrate reduction therapy in mouse models of the glycosphingolipidoses.
Tekijä: Platt, F, et al.
Julkaistu: (2003)

Substrate reduction therapy.
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Substrate reduction therapy for glycosphingolipid storage disorders.
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Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease.
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Substrate deprivation: a new therapeutic approach for the glycosphingolipid lysosomal storage diseases.
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Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.
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Julkaistu: (2008)

Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease
Tekijä: Ulrika Andersson, et al.
Julkaistu: (2004-08-01)

Substrate reduction therapy in the infantile form of Tay-Sachs disease.
Tekijä: Bembi, B, et al.
Julkaistu: (2006)

Substrate reduction therapy reduces brain ganglioside GM2 in neonatal Sandhoff disease mice
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Julkaistu: (2005)

Lucerastat, an iminosugar for substrate reduction therapy in Fabry disease: preclinical evidence
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Julkaistu: (2017)

Lucerastat, an iminosugar for substrate reduction therapy in Fabry Disease: Preclinical evidence
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Julkaistu: (2017)

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.
Tekijä: Jeyakumar, M, et al.
Julkaistu: (2001)

Substrate reduction therapy in Sandhoff disease: Evidence for improvement in nervous function in patients treated with miglustat
Tekijä: Lachmann, R, et al.
Julkaistu: (2006)

Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis.
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Julkaistu: (2002)

Imino sugar therapy for type 1 Gaucher disease.
Tekijä: Priestman, D, et al.
Julkaistu: (2000)

Substrate reduction therapy for inborn errors of metabolism
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Julkaistu: (2019)

Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy.
Tekijä: Platt, F, et al.
Julkaistu: (2001)

Miglustat - A viewpoint
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Julkaistu: (2003)

Glycolipid depletion in antimicrobial therapy.
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Julkaistu: (2003)

Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice
Tekijä: J.L. Kasperzyk, et al.
Julkaistu: (2005-04-01)

Eliglustat substrate reduction therapy in children with Gaucher disease type 1
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Julkaistu: (2025-02-01)

Effects of substrate deprivation therapy on brain lipids in neonatal GML storage disease mice
Tekijä: Kasperzyk, J, et al.
Julkaistu: (2003)

Iminosugar inhibitors for treating the lysosomal glycosphingolipidoses
Tekijä: Butters, T, et al.
Julkaistu: (2005)

Therapeutic applications of imino sugars in lysosomal storage disorders.
Tekijä: Butters, T, et al.
Julkaistu: (2003)

Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.
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Julkaistu: (2005)

New therapeutics for the treatment of glycosphingolipid lysosomal storage diseases.
Tekijä: Butters, T, et al.
Julkaistu: (2003)

Inhibition of glycosphingolipid biosynthesis: application to lysosomal storage disorders.
Tekijä: Butters, T, et al.
Julkaistu: (2000)

Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders
Tekijä: Maria Francisca Coutinho, et al.
Julkaistu: (2016-11-01)

"Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice" (vol 46, pg 744, 2004)
Tekijä: Kasperzyk, J, et al.
Julkaistu: (2005)

Substrate deprivation therapy reduces brain ganglioside content and GM1 in neonatal storage disease mice
Tekijä: Kasperzyk, J, et al.
Julkaistu: (2002)

Glycolipids in cell biology and medicine - Introduction
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Julkaistu: (2003)

Introduction
Tekijä: Dwek, R, et al.
Julkaistu: (2003)

Structure-based inhibitor design for substrate reduction therapy towards primary hyperoxaluria and galactosaemia
Tekijä: Mackinnon, S
Julkaistu: (2019)

Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.
Tekijä: John Marshall, et al.
Julkaistu: (2010-11-01)

Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease
Tekijä: Margarita M. Ivanova, et al.
Julkaistu: (2021-01-01)

Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease.
Tekijä: Margarita M Ivanova, et al.
Julkaistu: (2021-01-01)

Glycosylation: disease targets and therapy.
Tekijä: Zitzmann, N, et al.
Julkaistu: (2005)

Effect of Substrate Reduction Therapy in Comparison to Enzyme Replacement Therapy on Immune Aspects and Bone Involvement in Gaucher Disease
Tekijä: Renuka P. Limgala, et al.
Julkaistu: (2020-03-01)

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.
Tekijä: Cox, T, et al.
Julkaistu: (2000)

Erratum: Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice (Journal of Lipid Research (April 2004) 46 (744-751))
Tekijä: Kasperzyk, J, et al.
Julkaistu: (2005)