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公开(公告)号：US20180291376A1
公开(公告)日：2018-10-11
申请号：US15818073
申请日：2017-11-20
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113 , C12N15/11
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US09717750B2
公开(公告)日：2017-08-01
申请号：US14617388
申请日：2015-02-09
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： C07H21/02 , C07H21/04 , A61K31/70 , A61K31/7088 , C12N15/113 , C12Q1/68 , A61K31/712
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20190030058A1
公开(公告)日：2019-01-31
申请号：US15892560
申请日：2018-02-09
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/7088 , C12N15/113 , A61K31/712
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20240238326A1
公开(公告)日：2024-07-18
申请号：US18494445
申请日：2023-10-25
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/7088 , C12N15/113 , A61K31/712
CPC分类号： A61K31/7088 , C12N15/113 , A61K31/712 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20200376018A1
公开(公告)日：2020-12-03
申请号：US16708739
申请日：2019-12-10
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/7088 , C12N15/113
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20170088835A1
公开(公告)日：2017-03-30
申请号：US15267408
申请日：2016-09-16
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3341 , C12N2320/33 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20200181613A1
公开(公告)日：2020-06-11
申请号：US16507985
申请日：2019-07-10
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113 , C12N15/11
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US20150353929A1
公开(公告)日：2015-12-10
申请号：US14584112
申请日：2014-12-29
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3341 , C12N2320/33 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
摘要翻译： 本文公开了用于调节细胞，组织或动物中SMN2mRNA的剪接的化合物，组合物和方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。
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公开(公告)号：US08946183B2
公开(公告)日：2015-02-03
申请号：US13720474
申请日：2012-12-19
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C07H21/02 , C07H21/04 , A61K31/70 , C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3341 , C12N2320/33 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
摘要翻译： 本文公开了用于调节细胞，组织或动物中SMN2mRNA的剪接的化合物，组合物和方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。
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公开(公告)号：US20130109091A1
公开(公告)日：2013-05-02
申请号：US13720474
申请日：2012-12-19
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3341 , C12N2320/33 , C12N2310/3525
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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