Treatment of diseases associated with hepatic stellate cell activation using ammonia-lowering therapies
US-10525029-B2 · Jan 7, 2020 · US
US11083702B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11083702-B2 |
| Application number | US-201716094828-A |
| Country | US |
| Kind code | B2 |
| Filing date | Apr 18, 2017 |
| Priority date | Apr 19, 2016 |
| Publication date | Aug 10, 2021 |
| Grant date | Aug 10, 2021 |
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Methods of treating propionic acidemia (PA), methylmalonic acidemia (MMA) and fatty acid oxidation disorders are described. The methods include administering an anaplerotic agent that can directly enter the tricarboxylic acid cycle, such as a succinate derivative or pro-drug, for example trisuccinylglycerol (TSG). Methods of restoring tricarboxylic acid (TCA) cycle function in a cell deficient for propionyl-CoA carboxylase (PCC) or methylmalonyl-CoA mutase (MUT) by contacting the cell with a succinate derivative or pro-drug, such as TSG, are also described.
Opening claim text (preview).
The invention claimed is: 1. A method of treating propionic (PA) or methylmalonic acidemia (MMA) in a subject, comprising administering to the subject a therapeutically effective amount of trisuccinylglycerol
Esters of carboxylic acids having esterified carboxylic groups bound to acyclic carbon atoms and having any of the groups OH, O—metal, —CHO, keto, ether, acyloxy, [IMAGE cpc-sch-C07C-0958.gif] groups,[IMAGE cpc-sch-C07C-0959.gif] groups, or[IMAGE cpc-sch-C07C-0960.gif] in the acid moiety · CPC title
Polycarboxylic acids · CPC title
Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) · CPC title
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