JP2022526643A - 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 - Google Patents

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 Download PDF

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JP2022526643A
JP2022526643A JP2021559779A JP2021559779A JP2022526643A JP 2022526643 A JP2022526643 A JP 2022526643A JP 2021559779 A JP2021559779 A JP 2021559779A JP 2021559779 A JP2021559779 A JP 2021559779A JP 2022526643 A JP2022526643 A JP 2022526643A
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JP2022526643A5 (https=
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コング ロナルド
ウィリアム マッキントッシュ ジョセフ
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PTC Therapeutics Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245—Oxadiazoles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants

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  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Biomedical Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
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

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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

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

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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

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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)

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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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006522826A (ja) * 2003-04-11 2006-10-05 ピーティーシー セラピューティクス，インコーポレーテッド １，２，４−オキサジアゾール安息香酸化合物ならびにナンセンス抑制及び疾患の治療のためのその使用
JP2008535846A (ja) * 2005-04-08 2008-09-04 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異抑制治療用の、経口的に活性な１，２，４−オキサジアゾール組成物
JP2009532368A (ja) * 2006-03-30 2009-09-10 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異を有するｄｎａからの機能的タンパク質の産生のための方法及びそれと関連した障害の治療
JP2010506840A (ja) * 2006-10-12 2010-03-04 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異抑制治療用の経口的に活性な１，２，４−オキサジアゾールの投与方法

Family Cites Families (8)

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Publication number Priority date Publication date Assignee Title
DE102005061832A1 (de) 2005-12-23 2007-06-28 Patent-Treuhand-Gesellschaft für elektrische Glühlampen mbH Hochdruckentladungslampe mit verbesserter Zündfähigkeit sowie Hochspannungspulsgenerator
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
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006522826A (ja) * 2003-04-11 2006-10-05 ピーティーシー セラピューティクス，インコーポレーテッド １，２，４−オキサジアゾール安息香酸化合物ならびにナンセンス抑制及び疾患の治療のためのその使用
JP2008535846A (ja) * 2005-04-08 2008-09-04 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異抑制治療用の、経口的に活性な１，２，４−オキサジアゾール組成物
JP2009532368A (ja) * 2006-03-30 2009-09-10 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異を有するｄｎａからの機能的タンパク質の産生のための方法及びそれと関連した障害の治療
JP2010506840A (ja) * 2006-10-12 2010-03-04 ピーティーシー セラピューティクス，インコーポレーテッド ナンセンス突然変異抑制治療用の経口的に活性な１，２，４−オキサジアゾールの投与方法

Non-Patent Citations (3)

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Title
"ANNEX 1 SUMMARY OF PRODUCT CHARACTERISTICS", EUROPEAN MEICINES AGENCY [ONLINE], JPN6024017182, 20 March 2019 (2019-03-20), pages 1 - 39, ISSN: 0005546311 *
"History of Changes for Study: NCT02819557, Study of Ataluren in 2 to <5 Year-Old Males With Duchenne", CLINICALTRIALS.GOV ARCHIVE [ONLINE], vol. [retrieved on 2024-04-30], JPN6024017181, 24 January 2019 (2019-01-24), ISSN: 0005316824 *
PLOS ONE, vol. 8, no. 12, JPN6024017184, 2013, pages 81302, ISSN: 0005546312 *

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Publication number Publication date
CA3135641A1 (en) 2020-10-15
EP4473971A3 (en) 2025-02-19
EP3952867A1 (en) 2022-02-16
JP2025160227A (ja) 2025-10-22
MX2021012428A (es) 2022-01-19
CN114007608A (zh) 2022-02-01
IL287053A (en) 2021-12-01
US12458629B2 (en) 2025-11-04
WO2020210432A8 (en) 2021-10-28
EA202192767A1 (ru) 2021-12-29
KR20210151131A (ko) 2021-12-13
WO2020210432A1 (en) 2020-10-15
US20220175739A1 (en) 2022-06-09
EP4473971A2 (en) 2024-12-11
EP3952867A4 (en) 2023-01-11

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