Substituted pyrazolo[1,5-a]pyrazines for spinal muscular atrophy
摘要:
A prophylactic or therapeutic agent for spinal muscular atrophy according to the present invention includes a compound represented by the formula (I) or a salt thereof:
the variables are described herein.
Provided herein are certain compounds useful as HTT modulators. Such compound are useful in the treatment of Huntington's disease.
[EN] PRMT5 INHIBITORS<br/>[FR] INHIBITEURS DE PRMT5
申请人:MERCK SHARP & DOHME
公开号:WO2021126729A1
公开(公告)日:2021-06-24
The present invention provides a compound of Formula (I) (I) and the pharmaceutically acceptable salts, esters, and prodrugs thereof, which are PRMT5 inhibitors. Also provided are methods of making compounds of Formula I, pharmaceutical compositions comprising compounds of Formula I, and methods of using these compounds to treat cancer, sickle cell, and hereditary persistence of foetal hemoglobin (HPFH) mutations.
[EN] 5-PYRIMIDINECARBOXAMIDE DERIVATIVES AND METHODS OF USING THE SAME<br/>[FR] DÉRIVÉS D'ACÉTAMIDO-PHÉNYLTÉTRAZOLE ET LEURS MÉTHODES D'UTILISATION
申请人:[en]RGENTA THERAPEUTICS, INC.
公开号:WO2023102231A1
公开(公告)日:2023-06-08
The present disclosure relates to compounds of Formula (I): and to their prodrugs, pharmaceutically acceptable salts, pharmaceutical compositions, methods of use, and methods for their preparation. The compounds of the present disclosure may act as small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded genes, and methods of use of the compounds for modulating splicing and treating related diseases and conditions. The compounds disclosed herein may possess activity toward various genetic pathways and are accordingly useful in methods of treatment of the human or animal body.