METHODS OF TREATING DISORDERS ASSOCIATED WITH CASTOR

The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via sm...

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Hauptverfasser: JACKOWSKI, Suzanne, LEE, Richard, E, SUBRAMANIAN, Chitra, REDDY, T., Jagadeeswar, ROCK, Charles, O, EDWARDS, Anne, V, ZAMBONI, Robert, LIU, Jiuyu, YUN, Mi, Kyung, TANGALLAPALLY, Rajendra, P, SHARMA, Lalit, Kumar
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.