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
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
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 is an investigational, oral substrate reduction therapy for Gaucher disease type 1 (GD1). Nineteen treatment-naïve… (More)
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2014
2014
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
Mucopolysaccharidoses (MPS) are severe, inherited metabolic disorders caused by storage of glycosaminoglycans (GAGs). Sanfilippo… (More)
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2010
2010
Neurological pathology is characteristic of the mucopolysaccharidoses (MPSs) that store heparan sulphate (HS) glycosaminoglycan… (More)
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2010
2010
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 (SRT) is considered to be a potential therapeutic option for juvenile GM2 gangliosidosis (jGM2g). We… (More)
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2008
2008
BACKGROUND Mucopolysaccharidoses (MPSs) are a group of severe metabolic disorders caused by deficiencies in enzymes involved in… (More)
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2008
2008
GM1 gangliosidosis is an inherited neurodegenerative disorder caused by lysosomal beta-galactosidase deficiency, resulting in the… (More)
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