BR112014019750A8 - composto, composlção farmacêutlca, método para aumentar a lnclusão de éxon 7 de smn2 em mrna, método para aumentar a quantidade de proteína smn e método para tratar sma em um indivíduo humano em necessidade - Google Patents
composto, composlção farmacêutlca, método para aumentar a lnclusão de éxon 7 de smn2 em mrna, método para aumentar a quantidade de proteína smn e método para tratar sma em um indivíduo humano em necessidadeInfo
- Publication number
- BR112014019750A8 BR112014019750A8 BR112014019750A BR112014019750A BR112014019750A8 BR 112014019750 A8 BR112014019750 A8 BR 112014019750A8 BR 112014019750 A BR112014019750 A BR 112014019750A BR 112014019750 A BR112014019750 A BR 112014019750A BR 112014019750 A8 BR112014019750 A8 BR 112014019750A8
- Authority
- BR
- Brazil
- Prior art keywords
- increasing
- mrna
- inclusion
- compound
- pharmaceutical composition
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
- C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/04—Ortho-condensed systems
-
- 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
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
- C07D239/70—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings condensed with carbocyclic rings or ring systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
- Pyridine Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Steroid Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261597523P | 2012-02-10 | 2012-02-10 | |
| US61/597,523 | 2012-02-10 | ||
| PCT/US2013/025292 WO2013119916A2 (en) | 2012-02-10 | 2013-02-08 | Compounds for treating spinal muscular atrophy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| BR112014019750A2 BR112014019750A2 (pt) | 2017-06-20 |
| BR112014019750A8 true BR112014019750A8 (pt) | 2018-01-16 |
| BR112014019750B1 BR112014019750B1 (pt) | 2020-03-03 |
Family
ID=48948163
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112014019750-4A BR112014019750B1 (pt) | 2012-02-10 | 2013-02-08 | Composto, composição farmacêutica e seus usos |
Country Status (30)
| Country | Link |
|---|---|
| US (6) | US9586955B2 (pt) |
| EP (2) | EP2812004B1 (pt) |
| JP (2) | JP6092897B2 (pt) |
| KR (2) | KR102137087B1 (pt) |
| CN (2) | CN104349777B (pt) |
| AR (1) | AR092794A1 (pt) |
| AU (2) | AU2013216870B2 (pt) |
| BR (1) | BR112014019750B1 (pt) |
| CA (1) | CA2863874C (pt) |
| CL (1) | CL2014002100A1 (pt) |
| CO (1) | CO7061082A2 (pt) |
| CR (1) | CR20140376A (pt) |
| DK (1) | DK2812004T3 (pt) |
| EA (2) | EA029542B1 (pt) |
| EC (1) | ECSP14017269A (pt) |
| ES (1) | ES2697174T3 (pt) |
| HK (1) | HK1202077A1 (pt) |
| HU (1) | HUE039779T2 (pt) |
| IL (2) | IL233959A (pt) |
| MA (1) | MA35920B1 (pt) |
| MX (2) | MX354074B (pt) |
| NZ (1) | NZ628186A (pt) |
| PE (1) | PE20142364A1 (pt) |
| PH (2) | PH12014501786A1 (pt) |
| PL (1) | PL2812004T3 (pt) |
| SG (2) | SG10201609188WA (pt) |
| TR (1) | TR201813877T4 (pt) |
| TW (2) | TWI585085B (pt) |
| UA (1) | UA116981C2 (pt) |
| WO (1) | WO2013119916A2 (pt) |
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| EA028344B1 (ru) | 2012-01-26 | 2017-11-30 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения спинальной мышечной атрофии |
| UA116981C2 (uk) * | 2012-02-10 | 2018-06-11 | ПіТіСі ТЕРАП'ЮТІКС, ІНК. | Сполуки для лікування спінальної м'язової атрофії |
| JP6092264B2 (ja) | 2012-03-01 | 2017-03-08 | ピーティーシー セラピューティクス， インコーポレイテッド | 脊髄性筋委縮症を処置するための化合物 |
| KR102109992B1 (ko) | 2012-03-23 | 2020-05-13 | 피티씨 테라퓨틱스, 인크. | 척수성 근위축증을 치료하기 위한 화합물 |
| MD20150056A2 (ro) * | 2012-12-24 | 2015-09-30 | Ramot At Tel-Aviv University Ltd | Agenţi pentru tratarea bolilor genetice cauzate de mutaţii nonsens şi metode de identificare a acestora |
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| UA119670C2 (uk) | 2014-05-15 | 2019-07-25 | Ф. Хоффманн-Ля Рош Аг | Сполуки для лікування спінальної м'язової атрофії |
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| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
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| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
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