CN104244944B - 用于治疗脊髓性肌萎缩症的化合物 - Google Patents
用于治疗脊髓性肌萎缩症的化合物 Download PDFInfo
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- CN104244944B CN104244944B CN201280071056.5A CN201280071056A CN104244944B CN 104244944 B CN104244944 B CN 104244944B CN 201280071056 A CN201280071056 A CN 201280071056A CN 104244944 B CN104244944 B CN 104244944B
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- 0 CCC(CC1)C2C(CC)*C=CC(C)CCCC1C2 Chemical compound CCC(CC1)C2C(CC)*C=CC(C)CCCC1C2 0.000 description 17
- VJOUHFZHEUCANO-UHFFFAOYSA-N BCC(C1=CC(C=CC(C2)F)=C2OC1=O)=O Chemical compound BCC(C1=CC(C=CC(C2)F)=C2OC1=O)=O VJOUHFZHEUCANO-UHFFFAOYSA-N 0.000 description 1
- LSCHAZFMFBUCOV-CTZVLDLTSA-N CC(CC/C=C\C(N1CCNCC1)=C)N1N=C(C)SC1=N Chemical compound CC(CC/C=C\C(N1CCNCC1)=C)N1N=C(C)SC1=N LSCHAZFMFBUCOV-CTZVLDLTSA-N 0.000 description 1
- CERDICCHQPCUDZ-HWZXHQHMSA-N CC([C@](C)(C(CBr)=O)C(O)=O)F Chemical compound CC([C@](C)(C(CBr)=O)C(O)=O)F CERDICCHQPCUDZ-HWZXHQHMSA-N 0.000 description 1
- GLULNELZDJIROH-UHFFFAOYSA-N CC1=CN2C=C(C3=Cc4ccc(CN(C)CCO)cc4O/C3=[O]/C)NC2CC=C1 Chemical compound CC1=CN2C=C(C3=Cc4ccc(CN(C)CCO)cc4O/C3=[O]/C)NC2CC=C1 GLULNELZDJIROH-UHFFFAOYSA-N 0.000 description 1
- WPUZGDHERSSZGI-UHFFFAOYSA-N CC1C(C)=CN(C=C(C=C2)C3=Cc(ccc(CCCN(C)C)c4)c4OC3=O)C2=CC1 Chemical compound CC1C(C)=CN(C=C(C=C2)C3=Cc(ccc(CCCN(C)C)c4)c4OC3=O)C2=CC1 WPUZGDHERSSZGI-UHFFFAOYSA-N 0.000 description 1
- IUTIUNXOYCHQDT-UHFFFAOYSA-N CCC(C1)=CC=CC2=C1OCC(C(C=C)/N=C1\NC=C=C(C)CC=C1)=C2 Chemical compound CCC(C1)=CC=CC2=C1OCC(C(C=C)/N=C1\NC=C=C(C)CC=C1)=C2 IUTIUNXOYCHQDT-UHFFFAOYSA-N 0.000 description 1
- VCBBNHMMWJMKSQ-UHFFFAOYSA-N CCCNCCNc1ccc(C=C(c(cc2)n[n]2-c2ccccc2)C(O2)=O)c2c1 Chemical compound CCCNCCNc1ccc(C=C(c(cc2)n[n]2-c2ccccc2)C(O2)=O)c2c1 VCBBNHMMWJMKSQ-UHFFFAOYSA-N 0.000 description 1
- VYMJUZXFYAREJY-UHFFFAOYSA-N CCOC(Cc1nc(cccc2)c2[s]1)=O Chemical compound CCOC(Cc1nc(cccc2)c2[s]1)=O VYMJUZXFYAREJY-UHFFFAOYSA-N 0.000 description 1
- FXHRAKUEZPSMLJ-UHFFFAOYSA-N CN1CCNCCC1 Chemical compound CN1CCNCCC1 FXHRAKUEZPSMLJ-UHFFFAOYSA-N 0.000 description 1
- BSZKLVSTNGEDHN-UHFFFAOYSA-N Cc([s]1)n[n]2c1nc(C(C(Oc1c3)=O)=Cc1ccc3F)c2 Chemical compound Cc([s]1)n[n]2c1nc(C(C(Oc1c3)=O)=Cc1ccc3F)c2 BSZKLVSTNGEDHN-UHFFFAOYSA-N 0.000 description 1
- HMPUHXCGUHDVBI-UHFFFAOYSA-N Cc1nnc(N)[s]1 Chemical compound Cc1nnc(N)[s]1 HMPUHXCGUHDVBI-UHFFFAOYSA-N 0.000 description 1
- RAIPHJJURHTUIC-UHFFFAOYSA-N Nc1ncc[s]1 Chemical compound Nc1ncc[s]1 RAIPHJJURHTUIC-UHFFFAOYSA-N 0.000 description 1
- JJHPPFYPLSFKPO-UHFFFAOYSA-N OCC#Cc1ccc(C=CC(O2)=O)c2c1 Chemical compound OCC#Cc1ccc(C=CC(O2)=O)c2c1 JJHPPFYPLSFKPO-UHFFFAOYSA-N 0.000 description 1
Classifications
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- 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
-
- 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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D231/00—Heterocyclic compounds containing 1,2-diazole or hydrogenated 1,2-diazole rings
- C07D231/02—Heterocyclic compounds containing 1,2-diazole or hydrogenated 1,2-diazole rings not condensed with other rings
- C07D231/10—Heterocyclic compounds containing 1,2-diazole or hydrogenated 1,2-diazole rings not condensed with other rings having two or three double bonds between ring members or between ring members and non-ring members
- C07D231/12—Heterocyclic compounds containing 1,2-diazole or hydrogenated 1,2-diazole rings not condensed with other rings having two or three double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D311/00—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
- C07D311/02—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings ortho- or peri-condensed with carbocyclic rings or ring systems
- C07D311/04—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring
- C07D311/06—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2
- C07D311/08—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2 not hydrogenated in the hetero ring
- C07D311/16—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2 not hydrogenated in the hetero ring substituted in position 7
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D311/00—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
- C07D311/02—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings ortho- or peri-condensed with carbocyclic rings or ring systems
- C07D311/04—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring
- C07D311/06—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2
- C07D311/08—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2 not hydrogenated in the hetero ring
- C07D311/18—Benzo[b]pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2 not hydrogenated in the hetero ring substituted otherwise than in position 3 or 7
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
- C07D405/04—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
- C07D405/12—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/14—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
- C07D413/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
- C07D417/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
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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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
- C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
- C07D495/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
- C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
- C07D513/04—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
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Animal Behavior & Ethology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
- Pyrane Compounds (AREA)
- Plural Heterocyclic Compounds (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161582064P | 2011-12-30 | 2011-12-30 | |
| US61/582,064 | 2011-12-30 | ||
| PCT/US2012/071899 WO2013101974A1 (en) | 2011-12-30 | 2012-12-28 | Compounds for treating spinal muscular atrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN104244944A CN104244944A (zh) | 2014-12-24 |
| CN104244944B true CN104244944B (zh) | 2018-06-08 |
Family
ID=48698624
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201280071056.5A Active CN104244944B (zh) | 2011-12-30 | 2012-12-28 | 用于治疗脊髓性肌萎缩症的化合物 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | USRE47689E1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2797592B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP6193881B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR102057351B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN104244944B (cg-RX-API-DMAC7.html) |
| BR (1) | BR112014016287B1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2861609C (cg-RX-API-DMAC7.html) |
| EA (1) | EA029984B1 (cg-RX-API-DMAC7.html) |
| MX (1) | MX352861B (cg-RX-API-DMAC7.html) |
| WO (1) | WO2013101974A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104203239B (zh) | 2012-01-26 | 2017-09-08 | Ptc医疗公司 | 用于治疗脊髓性肌萎缩的化合物 |
| ES2697174T3 (es) | 2012-02-10 | 2019-01-22 | Ptc Therapeutics Inc | Compuestos para tratar atrofia muscular espinal |
| US9371336B2 (en) | 2012-03-01 | 2016-06-21 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| MX358514B (es) | 2012-03-23 | 2018-08-24 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| 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 |
| CN103360269B (zh) * | 2013-07-16 | 2015-07-08 | 临海市联盛化学有限公司 | 一种3-氯-2-氨基苯酚的制备方法 |
| CA2915764A1 (en) | 2013-08-19 | 2015-02-26 | F. Hoffmann-La Roche Ag | Screening method |
| US10195202B2 (en) | 2013-12-19 | 2019-02-05 | Ptc Therapeutics, Inc. | Methods for modulating the amount of RNA transcripts |
| WO2015095449A1 (en) * | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount rna transcripts |
| JP6513105B2 (ja) | 2014-04-22 | 2019-05-15 | ウニヴェルズィテート バーゼル | トリアジン、ピリミジン及びピリジン誘導体の新規製造方法 |
| US10882868B2 (en) | 2014-05-15 | 2021-01-05 | Hoffmann-La Roche Inc. | Compounds for treating spinal muscular atrophy |
| RS59718B1 (sr) | 2014-05-15 | 2020-01-31 | Hoffmann La Roche | Jedinjenja za lečenje spinalne mišićne atrofije |
| MX375925B (es) * | 2014-06-25 | 2025-03-07 | Hoffmann La Roche | Compuestos imidazo[1,2-a]pirazin-1-il-benzamida para tratar atrofia muscular espinal. |
| CN104744376A (zh) * | 2015-03-14 | 2015-07-01 | 长沙深橙生物科技有限公司 | 一种2-异丙基嘧啶衍生物的制备方法 |
| CN104803921A (zh) * | 2015-03-14 | 2015-07-29 | 长沙深橙生物科技有限公司 | 一种苯取代嘧啶衍生物的制备方法 |
| EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| CA3002494C (en) | 2015-11-12 | 2021-01-12 | F. Hoffmann-La Roche Ag | Compositions for treating spinal muscular atrophy |
| AR106652A1 (es) * | 2015-11-12 | 2018-02-07 | Hoffmann La Roche | Compuestos para tratar la esclerosis lateral amiotrófica |
| WO2017087364A1 (en) * | 2015-11-16 | 2017-05-26 | Ptc Therapeutics, Inc. | Hydrogen isotope-enriched analogues of 1,2,4-oxandiazole benzoic acid compounds, compositions and uses thereof |
| EP3386978B1 (en) | 2015-12-10 | 2021-01-27 | H. Hoffnabb-La Roche Ag | Bridged piperidine derivatives |
| CN108697709A (zh) | 2015-12-10 | 2018-10-23 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| RU2018133288A (ru) * | 2016-02-23 | 2020-03-24 | Индиана Юниверсити Рисерч Энд Текнолоджи Корпорейшн | Комбинированные терапии для лечения спинальной мышечной атрофии |
| CN108779115B (zh) | 2016-10-14 | 2021-02-26 | 江苏恒瑞医药股份有限公司 | 五元杂芳环并桥环类衍生物、其制备方法及其在医药上的应用 |
| CN110352007A (zh) | 2016-11-28 | 2019-10-18 | Ptc医疗公司 | 用于调节rna剪接的方法 |
| WO2018226622A1 (en) | 2017-06-05 | 2018-12-13 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| CN111372611A (zh) | 2017-06-14 | 2020-07-03 | Ptc医疗公司 | 修饰rna剪接的方法 |
| BR112019027717A2 (pt) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics, Inc. | métodos para tratar a doença de huntington |
| EP3645121B8 (en) | 2017-06-28 | 2025-06-18 | PTC Therapeutics, Inc. | Methods for treating huntington's disease |
| IL272413B (en) | 2017-08-04 | 2022-09-01 | Skyhawk Therapeutics Inc | Methods and compositions for modulating splicing |
| CA3075968C (en) * | 2017-09-22 | 2023-04-04 | F. Hoffmann-La Roche Ag | Process for the preparation of 7-(4,7-diazaspiro[2,5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one derivatives |
| JP7590183B2 (ja) | 2018-03-13 | 2024-11-26 | 武田薬品工業株式会社 | 血漿カリクレインのインヒビターとしての置換されたイミダゾピリジン及びその使用 |
| CA3094703A1 (en) | 2018-03-27 | 2019-10-03 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| CN112805280B (zh) | 2018-06-27 | 2024-12-06 | Ptc医疗公司 | 用于治疗亨廷顿氏病的杂芳基化合物 |
| WO2020005882A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| JP7421507B2 (ja) | 2018-06-27 | 2024-01-24 | 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 | ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物 |
| US12012410B2 (en) | 2018-06-27 | 2024-06-18 | Reborna Biosciences, Inc. | Substituted pyrazolo[1,5-a]pyrazines for spinal muscular atrophy |
| WO2020146449A1 (en) * | 2019-01-08 | 2020-07-16 | Texas Tech University System | Small molecule analogs of the protein e4orf1 in the treatment and prevention of metabolic disorders |
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| BR112014016287A2 (pt) | 2017-07-04 |
| HK1202059A1 (en) | 2015-09-18 |
| JP2015504057A (ja) | 2015-02-05 |
| JP6193881B2 (ja) | 2017-09-06 |
| EP2797592B1 (en) | 2019-08-28 |
| BR112014016287B1 (pt) | 2022-08-09 |
| US9617268B2 (en) | 2017-04-11 |
| KR102057351B1 (ko) | 2019-12-18 |
| CA2861609A1 (en) | 2013-07-04 |
| EP2797592A4 (en) | 2016-01-13 |
| WO2013101974A1 (en) | 2013-07-04 |
| EA201491296A1 (ru) | 2015-01-30 |
| MX352861B (es) | 2017-12-13 |
| CA2861609C (en) | 2021-02-16 |
| US20150119380A1 (en) | 2015-04-30 |
| USRE47689E1 (en) | 2019-11-05 |
| CN104244944A (zh) | 2014-12-24 |
| WO2013101974A8 (en) | 2019-03-07 |
| EP2797592A1 (en) | 2014-11-05 |
| KR20140128310A (ko) | 2014-11-05 |
| MX2014007877A (es) | 2015-01-16 |
| EA029984B1 (ru) | 2018-06-29 |
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