FAQ

Substrate Deprivation Therapy

Known as: Substrate Reduction Therapy 
A therapeutic procedure based on the inhibition or reduction of the biosynthesis of compounds that cannot break down in the cells.
National Institutes of Health

Topic mentions per year

Topic mentions per year

2005-2018
0246820052018

Papers overview

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2015
2015
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
Fabry disease, an X-linked glycosphingolipid storage disorder, is caused by the deficient activity of α-galactosidase A (α-Gal A… (More)
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2014
2014
The phytoestrogen genistein modulates lysosomal metabolism and transcription factor EB (TFEB) activation.
Genistein (5,7-dihydroxy-3-(4-hydroxyphenyl)-4H-1-benzopyran-4-one) has been previously proposed as a potential drug for use in… (More)
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2014
2014
Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.
Eliglustat is an investigational, oral substrate reduction therapy for Gaucher disease type 1 (GD1). Nineteen treatment-naïve… (More)
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2014
2014
Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
Pompe disease is an autosomal recessive disorder caused by a deficiency of acid α-glucosidase (GAA; EC 3.2.1.20) and the… (More)
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2012
2012
The use of elevated doses of genistein-rich soy extract in the gene expression-targeted isoflavone therapy for Sanfilippo disease patients.
Mucopolysaccharidoses (MPS) are severe, inherited metabolic disorders caused by storage of glycosaminoglycans (GAGs). Sanfilippo… (More)
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2010
2010
Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses
Neurological pathology is characteristic of the mucopolysaccharidoses (MPSs) that store heparan sulphate (HS) glycosaminoglycan… (More)
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2010
2010
Therapeutic approaches for lysosomal storage diseases.
The lysosomal storage disorders (LSDs) comprise a heterogeneous group of inborn errors of metabolism characterized by tissue… (More)
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2009
2009
Substrate reduction therapy in juvenile GM2 gangliosidosis.
Substrate reduction therapy (SRT) is considered to be a potential therapeutic option for juvenile GM2 gangliosidosis (jGM2g). We… (More)
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2008
2008
Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.
BACKGROUND Mucopolysaccharidoses (MPSs) are a group of severe metabolic disorders caused by deficiencies in enzymes involved in… (More)
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2008
2008
Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.
GM1 gangliosidosis is an inherited neurodegenerative disorder caused by lysosomal beta-galactosidase deficiency, resulting in the… (More)
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