[EN] CERAMIDE GALACTOSYLTRANSFERASE INHIBITORS FOR THE TREATMENT OF DISEASE<br/>[FR] INHIBITEURS DE LA CÉRAMIDE GALACTOSYLTRANSFÉRASE POUR LE TRAITEMENT DE MALADIES
申请人:BIOMARIN PHARM INC
公开号:WO2019104011A1
公开(公告)日:2019-05-31
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme ceramide galactosyltransferase (CGT), such as, for example, lysosomal storage diseases. Examples of lysosomal storage diseases include, for example, Krabbe disease and Metachromatic Leukodystrophy.
本发明涉及化合物、制造此类化合物的方法、包含此类化合物的药物组合物和药剂,以及使用此类化合物治疗或预防与鞘氨醇半乳糖基转移酶(CGT)相关疾病或失调的方法,例如,例如,溶酶体贮积病。溶酶体贮积病的例子包括,例如,克拉伯病和异染性脑白质营养不良。