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    Therapeutic compositions and methods of treating glycolipid storage related disorders
    3.
    发明授权
    Therapeutic compositions and methods of treating glycolipid storage related disorders 有权
    治疗组合物和治疗糖脂存储相关疾病的方法

    公开(公告)号：US07348000B2

    公开(公告)日：2008-03-25

    申请号：US10758247

    申请日：2004-01-15

    申请人： Raymond A. Dwek ,  Terence D. Butters ,  Mylvaganam Jeyakumar ,  Frances Mary Platt ,  David Priestman

    发明人： Raymond A. Dwek ,  Terence D. Butters ,  Mylvaganam Jeyakumar ,  Frances Mary Platt ,  David Priestman

    IPC分类号： A61K38/47 ,  A01N43/40

    CPC分类号： A61K45/06

    摘要： A method for treating a glycolipid storage-related disorder, comprising administering a therapeutically effective amount of an inhibitor of glycolipid synthesis in combination with an agent capable of increasing the rate of glycolipid degradation or in combination with bone marrow transplantation. Inhibitors of glycolipid synthesis include N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGJ) or N-nonyldeoxynojirimycin (NN-DNJ). Glycolipid storage-related disorders include Gaucher disease, Sandhoff's disease, Fabry's disease, Tay-Sach's disease, Niemann-Pick C storage disease, GM1 gangliosidosis, genetic disorders in which neuronal glycolipid accumulation contributes to disease pathology.

    摘要翻译： 一种用于治疗糖脂存储相关病症的方法，包括给予治疗有效量的糖脂合成抑制剂与能够增加糖脂降解速率或与骨髓移植相结合的药剂的组合。 糖脂合成抑制剂包括N-丁基脱氧野尻霉素（NB-DNJ），N-丁基脱氧半乳糖吉非霉素（NB-DGJ）或N-壬基脱氧野尻霉素（NN-DNJ）。 糖脂存储相关疾病包括戈谢病，桑德霍夫病，法布里病，泰萨氏病，尼曼 - 皮克C储存病，GM1神经节苷脂病，遗传疾病，其中神经元糖脂积累有助于疾病病理学。

    Deoxynojirimycin and D-arabinitol analogs and methods of using
    6.
    发明授权
    Deoxynojirimycin and D-arabinitol analogs and methods of using 有权
    脱氧野尻霉素和D-阿拉伯糖醇类似物及其使用方法

    公开(公告)号：US08975280B2

    公开(公告)日：2015-03-10

    申请号：US11752015

    申请日：2007-05-22

    申请人： Terry D. Butters ,  Raymond A. Dwek ,  George W. J. Fleet

    发明人： Terry D. Butters ,  Raymond A. Dwek ,  George W. J. Fleet

    IPC分类号： A61K31/445 ,  C07D211/48 ,  C07D211/46

    CPC分类号： C07D211/48 ,  C07D211/46

    摘要： A compound of Formula I are provided: wherein R is: R1 is a substituted or unsubstituted alkyl group; W1-4 are independently selected from hydrogen, substituted or unsubstituted alkyl groups, substituted or unsubstituted haloalkyl groups, substituted or unsubstituted alkanoyl groups, substituted or unsubstituted aroyl groups, or substituted or unsubstituted haloalkanoyl groups; X1-5 are independently selected from H, NO2, N3, and NH2; Y is absent or is a substituted or unsubstituted C1-alkyl group, other than carbonyl; Z is selected from a bond or NH, provided that when Z is a bond, Y is absent, and provided that when Z is NH, Y is a substituted or unsubstituted C1-alkyl group, other than carbonyl. Also provided are D-arabinitol compounds, methods for preparing such compounds and compositions of such compounds, and methods of using such compounds.

    摘要翻译： 提供式I化合物：其中R是：R 1是取代或未取代的烷基; 取代或未取代的烷基，取代或未取代的烷酰基，取代或未取代的烷酰基，取代或未取代的芳酰基，或取代或未取代的卤代烷酰基; X1-5独立地选自H，NO2，N3和NH2; Y不存在或是除羰基以外的取代或未取代的C 1 - 烷基; Z选自键或NH，条件是当Z为键时，Y不存在，条件是当Z为NH时，Y为除羰基以外的取代或未取代的C 1 - 烷基。 还提供了D-阿拉伯糖醇化合物，制备这些化合物的方法和这些化合物的组合物，以及使用这些化合物的方法。