CN114007608A - 在儿科患者中治疗无义突变介导的杜氏肌营养不良症的方法 - Google Patents

在儿科患者中治疗无义突变介导的杜氏肌营养不良症的方法 Download PDF

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Publication number
CN114007608A
CN114007608A CN202080042488.8A CN202080042488A CN114007608A CN 114007608 A CN114007608 A CN 114007608A CN 202080042488 A CN202080042488 A CN 202080042488A CN 114007608 A CN114007608 A CN 114007608A
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China
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dose
patient
nmdmd
hours
aliskiren
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CN202080042488.8A
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English (en)
Chinese (zh)
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罗纳德·孔
约瑟夫·威廉·麦金托什
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PTC Therapeutics Inc
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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)
CN202080042488.8A 2019-04-10 2020-04-09 在儿科患者中治疗无义突变介导的杜氏肌营养不良症的方法 Pending CN114007608A (zh)

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

Publications (1)

Publication Number Publication Date
CN114007608A true CN114007608A (zh) 2022-02-01

Family

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Family Applications (1)

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CN202080042488.8A Pending CN114007608A (zh) 2019-04-10 2020-04-09 在儿科患者中治疗无义突变介导的杜氏肌营养不良症的方法

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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101896186A (zh) * 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US20160194630A1 (en) * 2013-09-04 2016-07-07 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
CN109152810A (zh) * 2016-01-06 2019-01-04 康德生物医疗技术公司 用于预防和治疗杜氏肌肉萎缩症的方法和组合物

Family Cites Families (9)

* 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
KR102497273B1 (ko) * 2014-03-06 2023-02-07 피티씨 테라퓨틱스, 인크. 1,2,4-옥사디아졸 벤조산의 약학 조성물 및 염

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101896186A (zh) * 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US20160194630A1 (en) * 2013-09-04 2016-07-07 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
CN109152810A (zh) * 2016-01-06 2019-01-04 康德生物医疗技术公司 用于预防和治疗杜氏肌肉萎缩症的方法和组合物

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
EUROPEAN MEDICINES AGENCY: "Translarna (ataluren)，EPAR-Medicine Overview", pages 5, Retrieved from the Internet <URL:https://www.ema.europa.eu/en/documents/overview/translarna-epar-medicine-overview_en.pdf> *
EUROPEAN MEDICINES AGENCY: "Translarna (ataluren)，Translarna-H-C-2720-II-0037 : EPAR-Assessment report-Variation", pages 12 - 13, Retrieved from the Internet <URL:https://www.ema.europa.eu/en/documents/variation-report/translarna-h-c-2720-ii-0037-epar-assessment-report-variation_en.pdf> *
EUROPEAN MEDICINES AGENCY: "Translarna (ataluren)，Translarna-H-C-2720-II-0049 : EPAR-Assessment Report-Variation", pages 10 - 11, Retrieved from the Internet <URL:https://www.ema.europa.eu/en/documents/variation-report/translarna-h-c-2720-ii-0049-epar-assessment-report-variation_en.pdf> *
MCDONALD CM等: "The Clinical Evaluator Training Group and the ACT DMD Study Group Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial", 《LANCET》, vol. 390, pages 1489 - 1498 *
RICHARD S FINKEL等: "Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy", 《PLOS ONE》, vol. 8, no. 12, pages 81302 *

Also Published As

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
JP2022526643A (ja) 2022-05-25
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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