[EN] COMBINATION THERAPY FOR TREATING MPS1<br/>[FR] POLYTHÉRAPIE POUR LE TRAITEMENT DE LA MPS1
申请人:GAIN THERAPEUTICS SA
公开号:WO2021156774A1
公开(公告)日:2021-08-12
The application is directed to compounds of formula (I) and their salts and solvates, wherein B, R1, R2, R3, R3', R4, R4', and R5 are as set forth in the specification, as well as to methods for their preparation, pharmaceutical compositions comprising the same, and use thereof for the treatment and/or prevention of, e.g., MPS1, optionally in combination with α-L-iduronidase or an analog or variant thereof, e.g., laronidase.
该申请涉及公式(I)化合物及其盐和溶剂化合物,其中B、R1、R2、R3、R3'、R4、R4'和R5如规范中所述,以及其制备方法、含有相同化合物的药物组合物,以及用于治疗和/或预防MPS1等疾病的方法,可选地与α-L-异硫酸酶或其类似物或变体(例如,laronidase)结合使用。