申请人:ICAGEN LLC
公开号:WO2021007350A1
公开(公告)日:2021-01-14
This disclosure relates to, in part, the treatment of an organic acidemia in a subject in need thereof via administration of a therapeutically effective amount of compounds that inhibit BCAT2. The disclosure also relates to, in part, methods for identifying a candidate compound for treatment of organic acidemias.
这份披露涉及部分通过给予抑制BCAT2的化合物的治疗有效量来治疗需要的受试者的有机酸血症。该披露还涉及部分用于识别治疗有机酸血症的候选化合物的方法。