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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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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：EP3013345B1
公开(公告)日：2017-11-22
申请号：EP14816651.5
申请日：2014-06-23
发明人： DAKKA, Amal , GREEN, Luke , KARP, Gary , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PINARD, Emmanuel , QI, Hongyan , RATNI, Hasane , RISHER, Nicole , WEETALL, Marla , WOLL, Matthew
IPC分类号： C07D263/56 , C07D413/12 , C07D471/04 , C07D487/04 , C07D487/10 , A61K31/497 , A61K31/4965 , A61P25/28
CPC分类号： C07D519/00 , C07D263/56 , C07D413/12 , C07D471/04 , C07D487/04 , C07D487/10
摘要： The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
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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：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：EP3544435A1
公开(公告)日：2019-10-02
申请号：EP17873550.2
申请日：2017-11-27
发明人： BHATTACHARYYA, Anuradha , DAKKA, Amal , EFFENBERGER, Kerstin , GABBETA, Vijayalakshmi , LI, Wencheng , NARYSHKIN, Nikolai , TROTTA, Christopher , WIEDINGER, Kari
IPC分类号： A01N43/90 , A61K31/519 , C07D239/70
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公开(公告)号：EP3013345A2
公开(公告)日：2016-05-04
申请号：EP14816651.5
申请日：2014-06-23
发明人： DAKKA, Amal , GREEN, Luke , KARP, Gary , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PINARD, Emmanuel , QI, Hongyan , RATNI, Hasane , RISHER, Nicole , WEETALL, Marla , WOLL, Matthew
IPC分类号： A61K31/497 , A61K31/4965 , C07D401/00
CPC分类号： C07D519/00 , C07D263/56 , C07D413/12 , C07D471/04 , C07D487/04 , C07D487/10
摘要： The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
摘要翻译： 本发明提供了式（I）的化合物及其药学上可接受的盐，其中A，B，X，Y，R 1和R 2如本文所述。 此外，本发明涉及式（I）化合物的制备，包含它们的药物组合物以及它们作为药物的用途。
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公开(公告)号：EP3310169A1
公开(公告)日：2018-04-25
申请号：EP16804178.8
申请日：2016-05-27
发明人： NARYSHKIN, Nikolai , DAKKA, Amal
IPC分类号： A01N43/90 , A61K31/519 , C07D239/70
CPC分类号： A61K48/0025 , A61K31/519 , A61K31/711 , C07D239/70 , C12N15/11 , C12N15/85 , C12N2320/33 , Y02A50/465
摘要： In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.
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公开(公告)号：EP3082820A1
公开(公告)日：2016-10-26
申请号：EP14877918.4
申请日：2014-12-18
发明人： NARYSHKIN, Nikolai
IPC分类号： A61K31/519 , C07D239/70 , A01N43/90
CPC分类号： A61K31/519 , A61K31/4545 , A61K31/496 , A61K31/5377 , A61K31/551 , C07D471/04 , C07D487/04 , C07D519/00
摘要： Described herein are methods for modulating the amount of a gene product and compounds for use in such methods. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product as the result of gene expression and compounds for use in such methods.
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