[EN] METHODS FOR TREATING NEUROLOGICAL SYMPTOMS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES<br/>[FR] MÉTHODES DE TRAITEMENT DE SYMPTÔMES NEUROLOGIQUES ASSOCIÉS À DES MALADIES LYSOSOMALES
申请人:GENZYME CORP
公开号:WO2021156769A1
公开(公告)日:2021-08-12
Methods are provided for treating or preventing neurological symptoms and disorders which are associated with, e.g., lysosomal storage diseases. The methods include enhancing neuronal connectivity within the brain of a subject, increasing brain tissue volume, or preventing or delaying loss of brain tissue volume in a subject. Also provided are methods for monitoring the progression or regression of a neurological disorder, or assessing the onset of a neurological disorder, associated with a lysosomal storage disease, in which brain tissue volume of the subject is measured.
[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。