JP2022526643A5 - - Google Patents

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Publication number
JP2022526643A5
JP2022526643A5 JP2021559779A JP2021559779A JP2022526643A5 JP 2022526643 A5 JP2022526643 A5 JP 2022526643A5 JP 2021559779 A JP2021559779 A JP 2021559779A JP 2021559779 A JP2021559779 A JP 2021559779A JP 2022526643 A5 JP2022526643 A5 JP 2022526643A5
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JP
Japan
Prior art keywords
dose
patient
hours
midday
evening
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021559779A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022526643A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/027382 external-priority patent/WO2020210432A1/en
Publication of JP2022526643A publication Critical patent/JP2022526643A/ja
Publication of JP2022526643A5 publication Critical patent/JP2022526643A5/ja
Priority to JP2025115231A priority Critical patent/JP2025160227A/ja
Pending legal-status Critical Current

Links

JP2021559779A 2019-04-10 2020-04-09 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法 Pending JP2022526643A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025115231A JP2025160227A (ja) 2019-04-10 2025-07-08 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962831931P 2019-04-10 2019-04-10
US62/831,931 2019-04-10
PCT/US2020/027382 WO2020210432A1 (en) 2019-04-10 2020-04-09 Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025115231A Division JP2025160227A (ja) 2019-04-10 2025-07-08 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法

Publications (2)

Publication Number Publication Date
JP2022526643A JP2022526643A (ja) 2022-05-25
JP2022526643A5 true JP2022526643A5 (https=) 2023-04-18

Family

ID=72751530

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021559779A Pending JP2022526643A (ja) 2019-04-10 2020-04-09 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法
JP2025115231A Pending JP2025160227A (ja) 2019-04-10 2025-07-08 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025115231A Pending JP2025160227A (ja) 2019-04-10 2025-07-08 小児患者においてナンセンス変異が介在するデュシェンヌ型筋ジストロフィーを治療するための方法

Country Status (10)

Country Link
US (1) US12458629B2 (https=)
EP (2) EP3952867A4 (https=)
JP (2) JP2022526643A (https=)
KR (1) KR20210151131A (https=)
CN (1) CN114007608A (https=)
CA (1) CA3135641A1 (https=)
EA (1) EA202192767A1 (https=)
IL (1) IL287053A (https=)
MX (1) MX2021012428A (https=)
WO (1) WO2020210432A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102665711B1 (ko) * 2021-12-21 2024-05-14 재단법인 아산사회복지재단 Ras 활성도를 이용한 신경섬유종증 치료제에 대한 감수성 예측용 바이오마커 조성물 및 Ras 활성 억제제를 포함하는 신경섬유종증 예방 또는 치료용 약학적 조성물
WO2025110781A1 (ko) * 2023-11-24 2025-05-30 재단법인 아산사회복지재단 Ampd3를 포함하는 신경섬유종증 치료제에 대한 감수성 예측용 바이오마커 조성물 및 ampd3 저해제를 포함하는 신경섬유종증 예방 또는 치료용 약학 조성물

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE047569T2 (hu) 2003-04-11 2020-04-28 Ptc Therapeutics Inc 1,2,4-oxadiazol-benzoesav vegyület és nonszensz szupresszióra történõ alkalmazása
PT2402002T (pt) 2005-04-08 2018-10-08 Ptc Therapeutics Inc Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense
DE102005061832A1 (de) 2005-12-23 2007-06-28 Patent-Treuhand-Gesellschaft für elektrische Glühlampen mbH Hochdruckentladungslampe mit verbesserter Zündfähigkeit sowie Hochspannungspulsgenerator
RU2462246C2 (ru) * 2006-03-30 2012-09-27 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы получения функционального белка из днк, имеющей нонсенс-мутацию, и лечения нарушений, ассоциированных с ней
MX2009002439A (es) 2006-09-08 2009-03-27 Ptc Therapeutics Inc Procesos para la preparacion de acidos 1,2,4-oxadiazolbenzoicos.
PL2076501T3 (pl) 2006-09-25 2016-06-30 Ptc Therapeutics Inc Postacie krystaliczne kwasu 3-[5-(2-fluorofenylo)[1,2,4]oksadiazol-3-ilo]-benzoesowego
US8101641B2 (en) 2006-09-25 2012-01-24 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof
RS54025B1 (sr) 2006-10-12 2015-10-30 Ptc Therapeutics Inc. Postupci doziranja oralno aktivnog 1,2,4-oksadiazola za supresionu terapiju nonsens mutacije
ES2639852T3 (es) * 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
EP3041958B1 (en) * 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
KR102497273B1 (ko) * 2014-03-06 2023-02-07 피티씨 테라퓨틱스, 인크. 1,2,4-옥사디아졸 벤조산의 약학 조성물 및 염
CN116196388A (zh) * 2016-01-06 2023-06-02 康德生物医疗有限公司 用于预防和治疗杜氏肌肉萎缩症的方法和组合物

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