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

Propionic acidemia (PA) and methylmalonic acidemia (MMA) are rare autosomal recessive disorders of propionyl-CoA (P-CoA) catabolism, caused by a deficiency in the enzymes P-CoA carboxylase and methylmalonyl-CoA (M-CoA) mutase, respectively. PA and MMA are classified as intoxication-type inborn error...

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Bibliographic Details

Published in:	Journal of medicinal chemistry Vol. 64; no. 8; p. 5037
Main Authors:	Armstrong, Allison J, Henke, Brad R, Collado, Maria Sol, Taylor, Justin M, Pourtaheri, Taylor D, Dillberger, John E, Roper, Thomas D, Wamhoff, Brian R, Olson, Matthew W, Figler, Robert A, Hoang, Stephen A, Reardon, John E, Johns, Brian A
Format:	Journal Article
Language:	English
Published:	United States 22.04.2021
ISSN:	1520-4804, 1520-4804
Online Access:	Get more information
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