[EN] METHODS FOR TREATING PROTEINOPATHIES<br/>[FR] PROCÉDÉS DE TRAITEMENT DE PROTÉINOPATHIES
申请人:GENZYME CORP
公开号:WO2016145046A1
公开(公告)日:2016-09-15
This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.
[EN] GLUCOSYLCERAMIDE SYNTHASE INHIBITORS<br/>[FR] INHIBITEURS DE SYNTHASE DE GLUCOSYLCÉRAMIDE
申请人:GENZYME CORP
公开号:WO2014043068A1
公开(公告)日:2014-03-20
The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
本发明涉及葡萄糖酰胺合成酶(GCS)的抑制剂,例如下图所示的式 I 化合物,如本文所定义的,可用于治疗代谢性疾病,如溶酶体贮积症,可单独使用或与酶替代疗法、囊性疾病联合使用,也可用于治疗癌症。