ncs-382 and Developmental-Disabilities

ncs-382 has been researched along with Developmental-Disabilities* in 2 studies

Reviews

1 review(s) available for ncs-382 and Developmental-Disabilities

Article	Year
Succinic semialdehyde dehydrogenase deficiency, a disorder of GABA metabolism: an update on pharmacological and enzyme-replacement therapeutic strategies. Journal of inherited metabolic disease, 2018, Volume: 41, Issue:4 We present an update to the status of research on succinic semialdehyde dehydrogenase (SSADH) deficiency (SSADHD), a rare disorder of GABA metabolism. This is an unusual disorder featuring the accumulation of both GABA and its neuromodulatory analog, gamma-hydroxybutyric acid (GHB), and recent studies have advanced the potential clinical application of NCS-382, a putative GHB receptor antagonist. Animal studies have provided proof-of-concept that enzyme replacement therapy could represent a long-term therapeutic option. The characterization of neuronal stem cells (NSCs) derived from aldehyde dehydrogenase 5a1 Topics: Amino Acid Metabolism, Inborn Errors; Animals; Benzocycloheptenes; Developmental Disabilities; Disease Models, Animal; Enzyme Replacement Therapy; GABA Antagonists; Humans; Mice; Mice, Knockout; Signal Transduction; Succinate-Semialdehyde Dehydrogenase; TOR Serine-Threonine Kinases	2018

Article

Year

Succinic semialdehyde dehydrogenase deficiency, a disorder of GABA metabolism: an update on pharmacological and enzyme-replacement therapeutic strategies.

Journal of inherited metabolic disease, 2018, Volume: 41, Issue:4

We present an update to the status of research on succinic semialdehyde dehydrogenase (SSADH) deficiency (SSADHD), a rare disorder of GABA metabolism. This is an unusual disorder featuring the accumulation of both GABA and its neuromodulatory analog, gamma-hydroxybutyric acid (GHB), and recent studies have advanced the potential clinical application of NCS-382, a putative GHB receptor antagonist. Animal studies have provided proof-of-concept that enzyme replacement therapy could represent a long-term therapeutic option. The characterization of neuronal stem cells (NSCs) derived from aldehyde dehydrogenase 5a1

Topics: Amino Acid Metabolism, Inborn Errors; Animals; Benzocycloheptenes; Developmental Disabilities; Disease Models, Animal; Enzyme Replacement Therapy; GABA Antagonists; Humans; Mice; Mice, Knockout; Signal Transduction; Succinate-Semialdehyde Dehydrogenase; TOR Serine-Threonine Kinases

2018

Other Studies

1 other study(ies) available for ncs-382 and Developmental-Disabilities

Article	Year
Toxicologic/transport properties of NCS-382, a γ-hydroxybutyrate (GHB) receptor ligand, in neuronal and epithelial cells: Therapeutic implications for SSADH deficiency, a GABA metabolic disorder. Toxicology in vitro : an international journal published in association with BIBRA, 2018, Volume: 46 Topics: Amino Acid Metabolism, Inborn Errors; Animals; Anticonvulsants; Benzocycloheptenes; Biomarkers; Cell Survival; Developmental Disabilities; Epithelial Cells; Gene Expression Regulation; Genotype; Humans; Mice; Mice, Knockout; Mitochondria; Neural Stem Cells; Neurons; Reactive Oxygen Species; Receptors, Cell Surface; Succinate-Semialdehyde Dehydrogenase; Superoxides	2018

Article

Year

Toxicologic/transport properties of NCS-382, a γ-hydroxybutyrate (GHB) receptor ligand, in neuronal and epithelial cells: Therapeutic implications for SSADH deficiency, a GABA metabolic disorder.

Toxicology in vitro : an international journal published in association with BIBRA, 2018, Volume: 46

Topics: Amino Acid Metabolism, Inborn Errors; Animals; Anticonvulsants; Benzocycloheptenes; Biomarkers; Cell Survival; Developmental Disabilities; Epithelial Cells; Gene Expression Regulation; Genotype; Humans; Mice; Mice, Knockout; Mitochondria; Neural Stem Cells; Neurons; Reactive Oxygen Species; Receptors, Cell Surface; Succinate-Semialdehyde Dehydrogenase; Superoxides

2018