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    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
    1.
    发明申请
    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES 审中-公开
    预防药物治疗疾病的方法

    公开(公告)号：US20150342940A1

    公开(公告)日：2015-12-03

    申请号：US14731603

    申请日：2015-06-05

    申请人： AMICUS THERAPEUTICS

    发明人： Elfrida BENJAMIN ,  Hung V. Do ,  Xiaoyang Wu ,  John Flanagan ,  Brandon Wustman

    IPC分类号： A61K31/445

    CPC分类号： A61K31/445 ,  C07K14/00 ,  C12Q1/34 ,  G01N33/6893 ,  G01N2333/47 ,  G01N2333/94 ,  G01N2800/38 ,  G01N2800/52

    摘要： The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.

    摘要翻译： 本发明提供了确定患有溶酶体储存障碍的患者是否将受益于用特定药物伴侣的治疗的方法。 本发明例举了在表达来自α-半乳糖苷酶A的突变体的细胞系中测定α-半乳糖苷酶A对药物分子伴侣如1-脱氧半乳糖吉非霉素的反应性的体外方法。本发明还提供了一种诊断患者法布里病的方法 怀疑患有法布里病。

    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
    3.
    发明申请
    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES 有权
    预防药物治疗疾病的方法

    公开(公告)号：US20140206721A1

    公开(公告)日：2014-07-24

    申请号：US14054369

    申请日：2013-10-15

    申请人： AMICUS THERAPEUTICS, INC.

    发明人： Elfrida Benjamin ,  Hung V. Do ,  Xiaoyang Wu ,  John Flanagan ,  Brandon Wustman

    IPC分类号： A61K31/445 ,  C12Q1/34

    CPC分类号： A61K31/445 ,  C07K14/00 ,  C12Q1/34 ,  G01N33/6893 ,  G01N2333/47 ,  G01N2333/94 ,  G01N2800/38 ,  G01N2800/52

    摘要： The present invention provides methods to determine whether a patient with a lysosomal storage disorder win benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.

    摘要翻译： 本发明提供了确定患有溶酶体储存障碍的患者是否获益于用特定药物分子伴侣治疗的方法。 本发明例举了在表达来自α-半乳糖苷酶A的突变体的细胞系中测定α-半乳糖苷酶A对药物分子伴侣如1-脱氧半乳糖吉非霉素的反应性的体外方法。本发明还提供了一种诊断患者法布里病的方法 怀疑患有法布里病。

    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
    5.
    发明申请
    METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES 审中-公开
    预防药物治疗疾病的方法

    公开(公告)号：US20170003301A1

    公开(公告)日：2017-01-05

    申请号：US15268662

    申请日：2016-09-19

    申请人： AMICUS THERAPEUTICS

    发明人： Elfrida BENJAMIN ,  Hung V. Do ,  Xiaoyang Wu ,  John Flanagan ,  Brandon Wustman

    IPC分类号： G01N33/68

    摘要： The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.

    摘要翻译： 本发明提供了确定患有溶酶体储存障碍的患者是否将受益于用特定药物伴侣的治疗的方法。 本发明例举了在表达来自α-半乳糖苷酶A的突变体的细胞系中测定α-半乳糖苷酶A对药物分子伴侣如1-脱氧半乳糖吉非霉素的反应性的体外方法。本发明还提供了一种诊断患者法布里病的方法 怀疑患有法布里病。