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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Vydané v:	Journal of medicinal chemistry Ročník 64; číslo 8; s. 5037
Hlavní autori:	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
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	United States 22.04.2021
ISSN:	1520-4804, 1520-4804
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