JP2015511224A5 - - Google Patents

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Publication number
JP2015511224A5
JP2015511224A5 JP2014554840A JP2014554840A JP2015511224A5 JP 2015511224 A5 JP2015511224 A5 JP 2015511224A5 JP 2014554840 A JP2014554840 A JP 2014554840A JP 2014554840 A JP2014554840 A JP 2014554840A JP 2015511224 A5 JP2015511224 A5 JP 2015511224A5
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JP
Japan
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alkyl
isochromen
amino
pyrazin
methylpiperazin
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JP2014554840A
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English (en)
Japanese (ja)
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JP2015511224A (ja
JP6193888B2 (ja
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Priority claimed from PCT/US2013/023067 external-priority patent/WO2013112788A1/en
Publication of JP2015511224A publication Critical patent/JP2015511224A/ja
Publication of JP2015511224A5 publication Critical patent/JP2015511224A5/ja
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Publication of JP6193888B2 publication Critical patent/JP6193888B2/ja
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JP2014554840A 2012-01-26 2013-01-25 脊髄性筋萎縮症を治療するための化合物 Active JP6193888B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261591102P 2012-01-26 2012-01-26
US61/591,102 2012-01-26
PCT/US2013/023067 WO2013112788A1 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017083760A Division JP2017171667A (ja) 2012-01-26 2017-04-20 脊髄性筋萎縮症を治療するための化合物

Publications (3)

Publication Number Publication Date
JP2015511224A JP2015511224A (ja) 2015-04-16
JP2015511224A5 true JP2015511224A5 (enExample) 2016-03-03
JP6193888B2 JP6193888B2 (ja) 2017-09-06

Family

ID=48873933

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2014554840A Active JP6193888B2 (ja) 2012-01-26 2013-01-25 脊髄性筋萎縮症を治療するための化合物
JP2017083760A Pending JP2017171667A (ja) 2012-01-26 2017-04-20 脊髄性筋萎縮症を治療するための化合物

Family Applications After (1)

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JP2017083760A Pending JP2017171667A (ja) 2012-01-26 2017-04-20 脊髄性筋萎縮症を治療するための化合物

Country Status (12)

Country Link
US (1) US9399649B2 (enExample)
EP (1) EP2809322B9 (enExample)
JP (2) JP6193888B2 (enExample)
KR (1) KR102064624B1 (enExample)
CN (1) CN104203239B (enExample)
BR (1) BR112014018027B1 (enExample)
CA (1) CA2862084C (enExample)
EA (1) EA028344B1 (enExample)
ES (1) ES2733644T3 (enExample)
MX (1) MX357834B (enExample)
PL (1) PL2809322T3 (enExample)
WO (1) WO2013112788A1 (enExample)

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JP6193881B2 (ja) 2011-12-30 2017-09-06 ピーティーシー セラピューティクス, インコーポレイテッド 脊髄性筋萎縮症を治療するための化合物
CA2862084C (en) * 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
ES2697174T3 (es) 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014102778A2 (en) * 2012-12-24 2014-07-03 Ramot At Tel-Aviv University Ltd. Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
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US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
DK3143025T3 (da) * 2014-05-15 2019-12-09 Hoffmann La Roche Forbindelser til behandling af spinal muskelatrofi
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
AU2016353961B2 (en) * 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
WO2017087486A1 (en) 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
KR20220100719A (ko) 2015-12-10 2022-07-15 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
WO2017097728A1 (en) 2015-12-10 2017-06-15 F. Hoffmann-La Roche Ag Bridged piperidine derivatives
JP6738044B2 (ja) * 2016-07-22 2020-08-12 Jnc株式会社 ベンゾピラン骨格を有する液晶性化合物、液晶組成物、および液晶表示素子
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
KR102636383B1 (ko) 2017-08-04 2024-02-14 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
CA3105174A1 (en) * 2018-06-27 2020-01-02 Reborna Biosciences, Inc. Prophylactic or therapeutic agent for spinal muscular atrophy
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
CN119320388A (zh) 2019-02-04 2025-01-17 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP7551629B2 (ja) 2019-02-05 2024-09-17 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
KR102913697B1 (ko) 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
JP7603594B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
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WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
JP6193881B2 (ja) 2011-12-30 2017-09-06 ピーティーシー セラピューティクス, インコーポレイテッド 脊髄性筋萎縮症を治療するための化合物
CA2862084C (en) * 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
ES2697174T3 (es) 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy

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