A novel small molecule approach for the treatment of propionic and ...

Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA) are inborn errors of metabolism affecting the catabolism of valine, isoleucine, ...

(PDF) A novel small molecule approach for the treatment of ...

The small molecule, HST5040, resulted in a dose-dependent reduction in the levels of P-CoA, M-CoA (in MMA) and the disease-relevant biomarkers ...

Identification of 2,2-Dimethylbutanoic Acid (HST5040), a Clinical Development Candidate for the Treatment of Propionic Acidemia and Methylmalonic Acidemia.

(Open Access) A novel small molecule approach for the treatment of ...

Mitochondrial disease, mitophagy, and cellular distress in methylmalonic acidemia. · Propionic acidemia in mice: Liver acyl-CoA levels and clinical course.

Isolated Methylmalonic Acidemia - GeneReviews® - NCBI Bookshelf

A novel small molecule approach for the treatment of propionic and methylmalonic acidemias. Mol Genet Metab 2021;133:71-82. [PMC free ...

MUT - Methylmalonyl-CoA mutase, mitochondrial - Homo sapiens ...

A novel small molecule approach for the treatment of propionic and methylmalonic acidemias. Armstrong A.J., Collado M.S., Henke B.R., Olson M.W., Hoang S.A. ...

Citations to Inhibition by propionyl-coenzyme A of N ... - JCI

2023 · A novel small molecule approach for the treatment of propionic and methylmalonic acidemias. AJ Armstrong, MS Collado, BR Henke, MW Olson, SA Hoang, CA ...

Relief of CoA sequestration and restoration of ...

A novel small molecule approach for the treatment of propionic and methyl- malonic acidemias. Mol Genet Metab. 2021;133:71-82. doi:10. 1016 ...

Disrupted propionate metabolism evokes transcriptional changes in ...

... propionic acidemia and investigated therapeutic ... A novel small molecule approach for the treatment of propionic and methylmalonic acidemias.

Small molecules targeting protein–protein interactions for cancer ...

Subsequently, a series of synthetic small molecule inhibitors showed promising in vitro activity, among which the DRI-C21045 inhibitor has good selectivity and ...

A Listing of Medical Journal Articles on Propionic Acidemia

A novel small molecule approach for the treatment of propionic and methylmalonic acidemias ... to treat using gene therapy. However, when ...

Understanding acute metabolic decompensation in propionic and ...

Identification of novel metabolite alterations ... Targeted analysis of amino acids revealed that histidine was significantly decreased in ( ...

Dual mRNA therapy restores metabolic function in long-term studies ...

We herein report an enzyme replacement approach to treat PA using a combination of two messenger RNAs (mRNAs) (dual mRNAs) encoding both human PCCA (hPCCA) and ...

Strategies for the development of stimuli-responsive small molecule ...

This review outlines strategies for designing stimuli-responsive small molecule prodrugs focused on cancer treatment, emphasizing their chemical structures.

Development of small molecule drugs targeting immune checkpoints

CA-170 is the first small molecule oral ICI to enter clinical trials. As reported in a phase I trial in 2016, CA-170 activates peripheral blood ...

Propionic Acidemia - an overview | ScienceDirect Topics

Treatment was designed to include a diet with low levels of amino acids isoleucine, threonine, valine, and methionine Addition of carnitine to the diet was also ...

Small Molecule Inhibitors Targeting Activator Protein 1 (AP-1)

Suto, M. J.; Ransone, L. J. Novel approaches for the treatment of inflammatory diseases: inhibitors of NF-kappaB and AP-1 Curr. Pharm. Des ...

Small Molecule Targeting Immune Cells: A Novel Approach ... - MDPI

To overcome the accumulative effect of conventional cancer treatment, immunotherapy emerged as the targeted strategy to combat advanced cancers. Broadly, ...

WO2017155890A1 - Novel small molecule antimicrobials

1. Preparation of Small Molecule Compounds. The small molecule solutions were prepared by dissolving 100 mg of each compound at room temperature in 1 ml DMSO in ...

Propionic Acidemia: Overview, Etiology and Pathophysiology ...

Messenger RNA (mRNA) therapeutics are under investigation for the treatment of a number of metabolic diseases caused by protein deficiency, ...