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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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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EA029155B1 (ru) 2012-03-01 2018-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX2015008146A (es) * 2012-12-24 2016-01-20 Univ Ramot Agentes para tratar enfermedades geneticas derivadas de mutaciones sin sentido y metodos para identificarlas.
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
WO2015095446A1 (en) * 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
DK3143025T3 (da) * 2014-05-15 2019-12-09 Hoffmann La Roche Forbindelser til behandling af spinal muskelatrofi
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
JP6659841B2 (ja) * 2015-11-12 2020-03-04 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 脊髄性筋萎縮症を処置するための組成物
CA3005434C (en) 2015-11-16 2024-09-10 Ohio State Innovation Foundation METHODS AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES USING MOTOR NERVE SURVIVAL PROTEIN (MNS)
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WO2018098446A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
BR112019026508A2 (pt) 2017-06-14 2020-07-14 Ptc Therapeutics, Inc. métodos para modificar o splicing do rna
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111163838B (zh) 2017-06-28 2023-03-28 Ptc医疗公司 用于治疗亨廷顿氏病的方法
RU2020105929A (ru) 2017-08-04 2021-09-06 Скайхоук Терапьютикс, Инк. Способы и композиции для модулирования сплайсинга
JP7399870B2 (ja) 2018-03-27 2023-12-18 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 ハンチントン病を処置するための化合物
EP3814360B8 (en) 2018-06-27 2024-11-06 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
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
PL3814357T3 (pl) 2018-06-27 2024-09-16 Ptc Therapeutics, Inc. Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona
EP3920926A4 (en) 2019-02-05 2022-10-19 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7603592B2 (ja) 2019-02-05 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
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN119638696A (zh) 2019-02-06 2025-03-18 斯基霍克疗法公司 用于调节剪接的方法和组合物
CN114245794B (zh) 2019-05-13 2024-09-13 Ptc医疗公司 用于治疗亨廷顿氏病的化合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN121488042A (zh) * 2023-06-09 2026-02-06 豪夫迈·罗氏有限公司 诱导型基因表达系统

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USRE47689E1 (en) 2011-12-30 2019-11-05 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
ES2733644T3 (es) * 2012-01-26 2019-12-02 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
JP6092897B2 (ja) 2012-02-10 2017-03-08 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
EA029155B1 (ru) 2012-03-01 2018-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy

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