CA3135641A1 - Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients - Google Patents

Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients Download PDF

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Publication number
CA3135641A1
CA3135641A1 CA3135641A CA3135641A CA3135641A1 CA 3135641 A1 CA3135641 A1 CA 3135641A1 CA 3135641 A CA3135641 A CA 3135641A CA 3135641 A CA3135641 A CA 3135641A CA 3135641 A1 CA3135641 A1 CA 3135641A1
Authority
CA
Canada
Prior art keywords
ataluren
dose
patient
age
nmdmd
Prior art date
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
CA3135641A
Other languages
English (en)
French (fr)
Inventor
Ronald Kong
Joseph William Mcintosh
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics Inc
Original Assignee
PTC Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Publication of CA3135641A1 publication Critical patent/CA3135641A1/en
Pending legal-status Critical Current

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Classifications

    • 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

Landscapes

  • 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)
CA3135641A 2019-04-10 2020-04-09 Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients Pending CA3135641A1 (en)

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
CA3135641A1 true CA3135641A1 (en) 2020-10-15

Family

ID=72751530

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3135641A Pending CA3135641A1 (en) 2019-04-10 2020-04-09 Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients

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

Also Published As

Publication number Publication date
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
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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