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公开(公告)号：EP2797592A1
公开(公告)日：2014-11-05
申请号：EP12862080.4
申请日：2012-12-28
发明人： WOLL, Matthew G. , CHEN, Guangming , CHOI, Soongyu , DAKKA, Amal , HUANG, Song , KARP, Gary Mitchell , LEE, Chang-Sun , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PAUSHKIN, Sergey , QI, Hongyan , TURPOFF, Anthony A. , WEETALL, Marla L. , WELCH, Ellen , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/353
CPC分类号： C07D487/04 , C07D231/12 , C07D311/16 , C07D311/18 , C07D405/04 , C07D405/12 , C07D405/14 , C07D413/04 , C07D413/14 , C07D417/04 , C07D417/14 , C07D471/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
摘要翻译： 本文提供了化合物，其组合物以及用于治疗脊髓性肌萎缩的用途。 在具体的实施方案中，本文提供了可用于调节SMN2的外显子7包含到从SMN2基因转录的mRNA中的形式的化合物。 在另一个具体的实施方案中，本文提供了可用于调节SMN1的外显子7包含到从SMN1基因转录的mRNA中的形式的化合物。 在又一个实施方案中，本文提供了可用于调节SMN1和SMN2的外显子7包含在分别从SMN1和SMN2基因转录的mRNA中的形式的化合物。
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公开(公告)号：EP3406252B1
公开(公告)日：2020-05-13
申请号：EP18179491.8
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai A. , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
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公开(公告)号：EP2797592B1
公开(公告)日：2019-08-28
申请号：EP12862080.4
申请日：2012-12-28
发明人： WOLL, Matthew G. , CHEN, Guangming , CHOI, Soongyu , DAKKA, Amal , HUANG, Song , KARP, Gary Mitchell , LEE, Chang-Sun , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PAUSHKIN, Sergey , QI, Hongyan , TURPOFF, Anthony A. , WEETALL, Marla L. , WELCH, Ellen , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/353 , C07D413/14 , C07D405/14 , C07D311/16 , C07D311/18 , C07D231/12 , C07D405/04 , C07D405/12 , C07D413/04 , C07D417/04 , C07D471/04 , C07D487/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00 , A61P25/00 , C07D417/14
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公开(公告)号：EP3406252A1
公开(公告)日：2018-11-28
申请号：EP18179491.8
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai A. , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
摘要： Provided herein are compounds of formula (IIa1), compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP2809322A1
公开(公告)日：2014-12-10
申请号：EP13740919.9
申请日：2013-01-25
发明人： CHEN, Guangming , DAKKA, Amal , KARP, Gary Mitchell , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , WEETALL, Marla L. , WELCH, Ellen , ZHAO, Xin , WEETAL, Marla L.
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP2812004B1
公开(公告)日：2018-06-27
申请号：EP13747100.9
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
CPC分类号： C07D471/04 , A61K31/519 , C07D239/70 , C07D487/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP2809322B9
公开(公告)日：2019-10-30
申请号：EP13740919.9
申请日：2013-01-25
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公开(公告)号：EP2809322B1
公开(公告)日：2019-05-15
申请号：EP13740919.9
申请日：2013-01-25
发明人： CHEN, Guangming , DAKKA, Amal , KARP, Gary Mitchell , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , WEETALL, Marla L. , WELCH, Ellen , ZHAO, Xin
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP2812004A2
公开(公告)日：2014-12-17
申请号：EP13747100.9
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A01N43/90 , A61K31/519 , C07D239/70
CPC分类号： C07D471/04 , A61K31/519 , C07D239/70 , C07D487/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
摘要翻译： 本文提供了化合物，其组合物以及用于治疗脊髓性肌萎缩的用途。
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