Disease models, In vitro pharmacology

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

Armstrong AJ, Henke BR, Collado MS, et al. April 13, 2021

Doi: 10.1021/acs.jmedchem.1c00124