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Use of the Novel Site-Directed Enzyme Enhancement Therapy (SEE-Tx) Drug Discovery Platform to Identify Pharmacological Chaperones for Glutaric Acidemia Type 1
Research ExplorerPublicationsUse of the Novel Site-Directed Enzyme Enhancement Therapy (S... Abstract Bibliographical data
Use of the Novel Site-Directed Enzyme Enhancement Therapy (SEE-Tx) Drug Discovery Platform to Identify Pharmacological Chaperones for Glutaric Acidemia Type 1
Introduction Click to copy section linkSection link copied! Glutaric acidemia type 1 (GA1, OMIM #231670) is a rare metabolic disorder caused by an inherited deficiency of the mitochondrial enzyme glutaryl-CoA dehydrogenase (GCDH), with an estimated prevalence ranging from 1 in 125,000 to 1 in 250,000 in newborns within high-risk populations.
Use of the Novel Site-Directed Enzyme Enhancement Therapy (SEE-Tx) Drug Discovery Platform to Identify Pharmacological Chaperones for Glutaric Acidemia Type 1
Introduction Click to copy section linkSection link copied! Glutaric acidemia type 1 (GA1, OMIM #231670) is a rare metabolic disorder caused by an inherited deficiency of the mitochondrial enzyme glutaryl-CoA dehydrogenase (GCDH), with an estimated prevalence ranging from 1 in 125,000 to 1 in 250,000 in newborns within high-risk populations.
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