[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.
[EN] HETEROCYCLIC TRPML1 AGONISTS AND USES THEREOF<br/>[FR] AGONISTES DE TRPML1 HÉTÉROCYCLIQUES ET LEURS UTILISATIONS
申请人:LIBRA THERAPEUTICS INC
公开号:WO2022240992A1
公开(公告)日:2022-11-17
Described herein are TRPML1inhibitors and pharmaceutical compositions comprising said inhibitors. The subject compounds and compositions are useful for the treatment of TRPML1-mediated disorders or diseases.
The new piperazine derivatives are modulators of TRPML and are useful in treating disorders related to TRPML activities such as lysosome storage diseases, muscular dystrophy, age-related common neurodegenerative diseases, oxidative stress or reactive oxygen species (ROS) related diseases, and ageing.