[EN] MODULATORS OF TRPML, THEIR COMPOSITIONS AND METHODS OF USE<br/>[FR] MODULATEURS DE CANAUX TRPML, LEURS COMPOSITIONS ET PROCÉDÉS D'UTILISATION
申请人:CARAWAY THERAPEUTICS INC
公开号:WO2022150461A1
公开(公告)日:2022-07-14
The present disclosure relates to methods of treating a ciliopathy, the method comprising administering to a patient in need thereof a therapeutically effective amount of a compound or pharmaceutical composition thereof, wherein the compound modulates a TRPML ion channel. The disclosure also relates to pharmaceutical compounds of Formula (III) and subformulas thereof, or a pharmaceutically acceptable salt or composition thereof, and methods of their use for modulating TRPML ion channels, and for treating disorders.
本公开涉及治疗纤毛病的方法,该方法包括向需要治疗的患者施用化合物或其制剂的治疗有效量,其中该化合物调节TRPML离子通道。本公开还涉及公式(III)及其亚式化合物的药物化合物,或其药学上可接受的盐或制剂,以及用于调节TRPML离子通道和治疗疾病的方法。