IL287053A - A method for the treatment of Duchenne muscular dystrophy-mediated nonsense mutation in medical patients - Google Patents
A method for the treatment of Duchenne muscular dystrophy-mediated nonsense mutation in medical patientsInfo
- Publication number
- IL287053A IL287053A IL287053A IL28705321A IL287053A IL 287053 A IL287053 A IL 287053A IL 287053 A IL287053 A IL 287053A IL 28705321 A IL28705321 A IL 28705321A IL 287053 A IL287053 A IL 287053A
- Authority
- IL
- Israel
- Prior art keywords
- treating
- muscular dystrophy
- duchenne muscular
- nonsense mutation
- pediatric patients
- Prior art date
Links
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 title 1
- 108020004485 Nonsense Codon Proteins 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 230000037434 nonsense mutation Effects 0.000 title 1
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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831931P | 2019-04-10 | 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 |
|---|---|
| IL287053A true IL287053A (en) | 2021-12-01 |
Family
ID=72751530
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL287053A IL287053A (en) | 2019-04-10 | 2021-10-06 | A method for the treatment of Duchenne muscular dystrophy-mediated nonsense mutation in medical 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)
| 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)
| 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 |
-
2020
- 2020-04-09 EP EP20788548.4A patent/EP3952867A4/en not_active Withdrawn
- 2020-04-09 KR KR1020217035972A patent/KR20210151131A/ko not_active Ceased
- 2020-04-09 CN CN202080042488.8A patent/CN114007608A/zh active Pending
- 2020-04-09 CA CA3135641A patent/CA3135641A1/en active Pending
- 2020-04-09 EA EA202192767A patent/EA202192767A1/ru unknown
- 2020-04-09 JP JP2021559779A patent/JP2022526643A/ja active Pending
- 2020-04-09 WO PCT/US2020/027382 patent/WO2020210432A1/en not_active Ceased
- 2020-04-09 US US17/602,576 patent/US12458629B2/en active Active
- 2020-04-09 EP EP24207847.5A patent/EP4473971A3/en active Pending
- 2020-04-09 MX MX2021012428A patent/MX2021012428A/es unknown
-
2021
- 2021-10-06 IL IL287053A patent/IL287053A/en unknown
-
2025
- 2025-07-08 JP JP2025115231A patent/JP2025160227A/ja active Pending
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 |
| CN114007608A (zh) | 2022-02-01 |
| 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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