A novel small molecule approach for the treatment of propionic and methylmalonic acidemias.
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DOI: 10.1016/j.ymgme.2021.03.001
Abstract: Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA) are inborn errors of metabolism affecting the catabolism of valine, isoleucine, methionine, threonine and odd-chain fatty acids. These are multi-organ disorders caused by the enzymatic deficiency of propionyl-CoA… read more here.
Keywords: hst5040; mma; small molecule; coa coa ... See more keywords