JP2022544538A5 - - Google Patents

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Publication number
JP2022544538A5
JP2022544538A5 JP2022509053A JP2022509053A JP2022544538A5 JP 2022544538 A5 JP2022544538 A5 JP 2022544538A5 JP 2022509053 A JP2022509053 A JP 2022509053A JP 2022509053 A JP2022509053 A JP 2022509053A JP 2022544538 A5 JP2022544538 A5 JP 2022544538A5
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JP
Japan
Prior art keywords
alkyl
substituted
alkoxy
independently selected
heteroaryl
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JP2022509053A
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English (en)
Japanese (ja)
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JP2022544538A (ja
JP7689110B2 (ja
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Priority claimed from PCT/US2020/046546 external-priority patent/WO2021030766A1/en
Publication of JP2022544538A publication Critical patent/JP2022544538A/ja
Publication of JP2022544538A5 publication Critical patent/JP2022544538A5/ja
Priority to JP2025087494A priority Critical patent/JP2025122671A/ja
Application granted granted Critical
Publication of JP7689110B2 publication Critical patent/JP7689110B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2022509053A 2019-08-15 2020-08-14 脊髄性筋萎縮症のための併用療法 Active JP7689110B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025087494A JP2025122671A (ja) 2019-08-15 2025-05-26 脊髄性筋萎縮症のための併用療法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887579P 2019-08-15 2019-08-15
US62/887,579 2019-08-15
PCT/US2020/046546 WO2021030766A1 (en) 2019-08-15 2020-08-14 Combination therapy for spinal muscular atrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025087494A Division JP2025122671A (ja) 2019-08-15 2025-05-26 脊髄性筋萎縮症のための併用療法

Publications (3)

Publication Number Publication Date
JP2022544538A JP2022544538A (ja) 2022-10-19
JP2022544538A5 true JP2022544538A5 (enExample) 2023-08-22
JP7689110B2 JP7689110B2 (ja) 2025-06-05

Family

ID=74570741

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022509053A Active JP7689110B2 (ja) 2019-08-15 2020-08-14 脊髄性筋萎縮症のための併用療法
JP2025087494A Pending JP2025122671A (ja) 2019-08-15 2025-05-26 脊髄性筋萎縮症のための併用療法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025087494A Pending JP2025122671A (ja) 2019-08-15 2025-05-26 脊髄性筋萎縮症のための併用療法

Country Status (4)

Country Link
US (1) US20220280548A1 (enExample)
EP (1) EP4013387A4 (enExample)
JP (2) JP7689110B2 (enExample)
WO (1) WO2021030766A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250206783A1 (en) * 2022-03-21 2025-06-26 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
WO2023202501A1 (zh) * 2022-04-18 2023-10-26 深圳市塔吉瑞生物医药有限公司 取代的吡啶并嘧啶酮类化合物及包含该化合物的组合物及其用途
JP2025534681A (ja) * 2022-10-14 2025-10-17 ジェネンテック， インコーポレイテッド 脊髄性筋萎縮症の治療方法
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115227709A (zh) * 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
JP2014533944A (ja) * 2011-11-11 2014-12-18 サンタリス ファーマ アー／エスＳａｎｔａｒｉｓ Ｐｈａｒｍａ Ａ／Ｓ Ｓｍｎ２スプライシングの調節のための化合物
KR20150030205A (ko) * 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
AU2016353961B2 (en) 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
AU2016370630B2 (en) * 2015-12-14 2023-04-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
ES2935658T3 (es) * 2016-07-15 2023-03-09 Ionis Pharmaceuticals Inc Compuestos y métodos para la modulación de SMN2
WO2020037161A1 (en) * 2018-08-15 2020-02-20 Biogen Ma Inc. Combination therapy for spinal muscular atrophy

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