JP2009541489A5 - - Google Patents
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- Publication number
- JP2009541489A5 JP2009541489A5 JP2009518489A JP2009518489A JP2009541489A5 JP 2009541489 A5 JP2009541489 A5 JP 2009541489A5 JP 2009518489 A JP2009518489 A JP 2009518489A JP 2009518489 A JP2009518489 A JP 2009518489A JP 2009541489 A5 JP2009541489 A5 JP 2009541489A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmacological chaperone
- glucocerebrosidase
- isophagomine
- disease
- individual
- 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.)
- Granted
Links
- 108010006519 Molecular Chaperones Proteins 0.000 claims 15
- 230000000144 pharmacologic effect Effects 0.000 claims 15
- 102000004547 Glucosylceramidase Human genes 0.000 claims 11
- 108010017544 Glucosylceramidase Proteins 0.000 claims 11
- 125000000217 alkyl group Chemical group 0.000 claims 6
- -1 isofagomine compound Chemical class 0.000 claims 6
- 230000004770 neurodegeneration Effects 0.000 claims 6
- 208000015122 neurodegenerative disease Diseases 0.000 claims 6
- 229910052760 oxygen Inorganic materials 0.000 claims 6
- 229910052717 sulfur Inorganic materials 0.000 claims 6
- 230000000694 effects Effects 0.000 claims 5
- 208000032859 Synucleinopathies Diseases 0.000 claims 4
- QPYJXFZUIJOGNX-HSUXUTPPSA-N afegostat Chemical class OC[C@H]1CNC[C@@H](O)[C@@H]1O QPYJXFZUIJOGNX-HSUXUTPPSA-N 0.000 claims 4
- 229910052799 carbon Inorganic materials 0.000 claims 4
- 229910052739 hydrogen Inorganic materials 0.000 claims 4
- 239000001257 hydrogen Substances 0.000 claims 4
- 229910052757 nitrogen Inorganic materials 0.000 claims 4
- 208000009829 Lewy Body Disease Diseases 0.000 claims 3
- 201000002832 Lewy body dementia Diseases 0.000 claims 3
- 208000018737 Parkinson disease Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000003112 inhibitor Substances 0.000 claims 3
- ULBPPCHRAVUQMC-MUMXBIPUSA-N (2r,3r)-2,3-dihydroxybutanedioic acid;(3r,4r,5r)-5-(hydroxymethyl)piperidine-3,4-diol Chemical group OC[C@H]1CNC[C@@H](O)[C@@H]1O.OC(=O)[C@H](O)[C@@H](O)C(O)=O ULBPPCHRAVUQMC-MUMXBIPUSA-N 0.000 claims 2
- 125000006201 3-phenylpropyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])([H])C([H])([H])* 0.000 claims 2
- 102000006378 Catechol O-methyltransferase Human genes 0.000 claims 2
- 108020002739 Catechol O-methyltransferase Proteins 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 125000004946 alkenylalkyl group Chemical group 0.000 claims 2
- 125000004183 alkoxy alkyl group Chemical group 0.000 claims 2
- 125000003545 alkoxy group Chemical group 0.000 claims 2
- 125000003118 aryl group Chemical group 0.000 claims 2
- 125000005242 carbamoyl alkyl group Chemical group 0.000 claims 2
- 230000002860 competitive effect Effects 0.000 claims 2
- 150000001875 compounds Chemical class 0.000 claims 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 125000001188 haloalkyl group Chemical group 0.000 claims 2
- 125000001475 halogen functional group Chemical group 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 150000002431 hydrogen Chemical class 0.000 claims 2
- 229910052738 indium Inorganic materials 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 229940098778 Dopamine receptor agonist Drugs 0.000 claims 1
- WTDRDQBEARUVNC-LURJTMIESA-N L-DOPA Chemical group OC(=O)[C@@H](N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-LURJTMIESA-N 0.000 claims 1
- WTDRDQBEARUVNC-UHFFFAOYSA-N L-Dopa Natural products OC(=O)C(N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-UHFFFAOYSA-N 0.000 claims 1
- 229940123685 Monoamine oxidase inhibitor Drugs 0.000 claims 1
- 229940121948 Muscarinic receptor antagonist Drugs 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 102000003802 alpha-Synuclein Human genes 0.000 claims 1
- 108090000185 alpha-Synuclein Proteins 0.000 claims 1
- 239000002260 anti-inflammatory agent Substances 0.000 claims 1
- 229940121363 anti-inflammatory agent Drugs 0.000 claims 1
- 239000000812 cholinergic antagonist Substances 0.000 claims 1
- 239000003954 decarboxylase inhibitor Substances 0.000 claims 1
- 239000003136 dopamine receptor stimulating agent Substances 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 229960004502 levodopa Drugs 0.000 claims 1
- 239000002899 monoamine oxidase inhibitor Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81595206P | 2006-06-23 | 2006-06-23 | |
| US60/815,952 | 2006-06-23 | ||
| PCT/US2007/072016 WO2007150064A2 (en) | 2006-06-23 | 2007-06-25 | METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2009541489A JP2009541489A (ja) | 2009-11-26 |
| JP2009541489A5 true JP2009541489A5 (cg-RX-API-DMAC7.html) | 2010-08-12 |
| JP5303458B2 JP5303458B2 (ja) | 2013-10-02 |
Family
ID=38834440
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009518489A Active JP5303458B2 (ja) | 2006-06-23 | 2007-06-25 | β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US7829579B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2040548B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP5303458B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR101402554B1 (cg-RX-API-DMAC7.html) |
| AT (1) | ATE555788T1 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2007260812B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BRPI0713442A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2656643C (cg-RX-API-DMAC7.html) |
| DK (1) | DK2040548T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2387845T3 (cg-RX-API-DMAC7.html) |
| IL (1) | IL196151A (cg-RX-API-DMAC7.html) |
| MX (1) | MX2009000032A (cg-RX-API-DMAC7.html) |
| PT (1) | PT2040548E (cg-RX-API-DMAC7.html) |
| WO (1) | WO2007150064A2 (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA200900270B (cg-RX-API-DMAC7.html) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| BRPI0713442A2 (pt) | 2006-06-23 | 2012-03-06 | Amicus Therapeutics, Inc. | Método para o tratamento de desrodens neurológicas, pelo aumento da atividade de b-glicocerebrosidase |
| GB0622702D0 (en) * | 2006-11-15 | 2006-12-27 | Univ Dundee | Selective glycosidase inhibitors |
| CN101754682B (zh) * | 2007-05-16 | 2014-11-12 | 布里格姆妇女医院 | 突触核蛋白病的治疗 |
| WO2009155936A1 (en) * | 2008-06-26 | 2009-12-30 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
| GB2480159A (en) * | 2008-11-14 | 2011-11-09 | Parkinson S Inst | Compositions and methods for the treatment of altered a-synuclein function |
| ES2505243T3 (es) * | 2009-04-09 | 2014-10-09 | Amicus Therapeutics, Inc. | Agentes terapéuticos para prevenir y/o tratar trastornos degenerativos del sistema nervioso central |
| ES2564093T3 (es) * | 2009-04-09 | 2016-03-17 | Amicus Therapeutics, Inc. | Métodos para prevenir y/o tratar trastornos del almacenamiento lisosomal |
| GB0906159D0 (en) * | 2009-04-09 | 2009-05-20 | Summit Corp Plc | Drug combination for the treatment of proteostatic diseases |
| EP2435459B1 (en) * | 2009-05-26 | 2016-05-04 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase |
| US9206457B2 (en) * | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| CN109172813B (zh) * | 2009-07-28 | 2022-09-02 | 武田药品工业株式会社 | 用于治疗戈谢病的组合物和方法 |
| ES2646099T3 (es) * | 2009-10-19 | 2017-12-12 | Amicus Therapeutics, Inc. | Método para el tratamiento de la enfermedad de Alzheimer utilizando chaperonas farmacológicas para aumentar la actividad de gangliosidasas |
| DK2490532T3 (en) * | 2009-10-19 | 2017-02-27 | Amicus Therapeutics Inc | Hitherto UNKNOWN COMPOSITIONS FOR PREVENTION AND / OR TREATMENT OF DEGENERATIVE DISORDERS IN THE CENTRAL Nervous System |
| LT2995306T (lt) * | 2009-10-19 | 2019-04-10 | Amicus Therapeutics, Inc. | Naujos kompozicijos lizosomų kaupimosi sutrikimų profilaktikai ir (arba) gydymui |
| PT2646044T (pt) | 2010-11-30 | 2019-11-12 | Orphazyme As | Métodos para aumentar a atividade intracelular de hsp70 |
| HRP20190867T1 (hr) | 2011-05-12 | 2019-07-12 | Proteostasis Therapeutics, Inc. | Regulatori proteostaze |
| EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
| JP6061922B2 (ja) * | 2011-06-22 | 2017-01-18 | ザ ジェネラル ホスピタル コーポレイション | プロテイノパチーの処置方法 |
| EP2806875B1 (en) | 2012-01-25 | 2017-07-19 | Proteostasis Therapeutics, Inc. | Proteasome activity modulating compounds |
| MX2015005722A (es) * | 2012-11-05 | 2016-01-12 | Genzyme Corp | Composiciones y metodos para el tratamiento de proteinopatias. |
| US9750712B2 (en) | 2012-12-07 | 2017-09-05 | Rush University Medical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| EP3087056A4 (en) | 2013-12-23 | 2017-07-19 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
| US9675627B2 (en) | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| KR20250069686A (ko) | 2014-09-15 | 2025-05-19 | 제브라 덴마크 에이/에스 | 아리모클로몰 제제 |
| WO2016073895A1 (en) | 2014-11-06 | 2016-05-12 | Lysosomal Therapeutics Inc. | Substituted pyrazolo(1,5-a)pyrimidines and their use in the treatment of medical disorders |
| US10967073B2 (en) | 2015-05-07 | 2021-04-06 | The Mclean Hospital Corporation | Glucocerebrosidase gene therapy for Parkinson's disease |
| WO2017176961A1 (en) | 2016-04-06 | 2017-10-12 | Lysosomal Therapeutics Inc. | Imidazo [1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders |
| JP7034935B2 (ja) * | 2016-04-06 | 2022-03-14 | リソソーマル・セラピューティクス・インコーポレイテッド | ピロロ［１，２－ａ］ピリミジニルカルボキサミド化合物および医学的障害の処置におけるその使用 |
| CA3020287A1 (en) | 2016-04-06 | 2017-10-12 | Lysosomal Therapeutics Inc. | Pyrazolo[1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders |
| WO2017178029A1 (en) | 2016-04-13 | 2017-10-19 | Orphazyme Aps | Heat shock proteins and cholesterol homeostasis |
| CA3287846A1 (en) | 2016-04-29 | 2025-11-29 | Zevra Denmark A/S | Arimoclomol for treating glucocerebrosidase associated disorders |
| EP3452481A4 (en) | 2016-05-05 | 2020-02-26 | Lysosomal Therapeutics Inc. | IMIDAZO [1,2-B] SUBSTITUTED PYRIDAZINES, IMIDAZO [1,5-B] SUBSTITUTED PYRIDAZINES, RELATED COMPOUNDS AND THEIR USE IN THE TREATMENT OF MEDICAL CONDITIONS |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| EP3700526A4 (en) * | 2017-10-26 | 2021-09-08 | Takeda Pharmaceutical Company Limited | FORMULATIONS WITH GLUCOCEREBROSIDASE AND ISOFAGOMIN |
| CN108428452B (zh) | 2018-03-14 | 2019-12-13 | 百度在线网络技术（北京）有限公司 | 终端支架和远场语音交互系统 |
| AU2019295765B2 (en) | 2018-06-27 | 2024-08-29 | Yumanity, Inc. | Proteasome activity enhancing compounds |
| WO2020006269A1 (en) | 2018-06-27 | 2020-01-02 | Proteostasis Therapeutics, Inc. | Proteasome activity enhancing compounds |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| JP7712673B2 (ja) * | 2018-12-05 | 2025-07-24 | ワシントン・ユニバーシティ | 神経の疾患を検出し、予防し、回復に向かわせ、治療する方法 |
| CN113906012A (zh) * | 2019-04-25 | 2022-01-07 | 武田药品工业有限公司 | 异法戈明盐、使用方法和调配物 |
| CN111450306B (zh) * | 2020-03-06 | 2021-11-19 | 大连理工大学 | 外用纳米羟基磷灰石/聚多巴胺湿黏附型止血粉及其制备方法 |
| AU2021380947C1 (en) | 2020-11-19 | 2025-02-20 | Zevra Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
| AU2022265691A1 (en) * | 2021-04-30 | 2023-11-09 | Vanqua Bio, Inc. | Small molecule modulators of glucocerebrosidase activity and uses thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ335290A (en) * | 1996-10-15 | 2001-08-31 | D Gen Ltd | Diagnosis an typing of spongiform encephalopathy (or bovine spongiform encephalopathy (BSE)) |
| GB2355074A (en) | 1996-10-15 | 2001-04-11 | Imperial College | Method for predicting susceptibility to bovine spongiform encephalopathy |
| US6274597B1 (en) * | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| EP2444102B1 (en) * | 2003-01-31 | 2015-04-08 | The Mount Sinai School of Medicine of New York University | Combination therapy for treating protein deficiency disorders |
| US20050137223A1 (en) | 2003-11-12 | 2005-06-23 | Amicus Therapeutics, Inc. | Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease |
| CN104771402A (zh) | 2005-06-08 | 2015-07-15 | 阿米库斯治疗学公司 | 溶酶体酶编码基因突变相关的cns紊乱的治疗 |
| PL1860101T3 (pl) * | 2006-05-24 | 2011-10-31 | Amicus Therapeutics Inc | Sól winianu izofagominy i sposoby zastosowania |
| BRPI0713442A2 (pt) | 2006-06-23 | 2012-03-06 | Amicus Therapeutics, Inc. | Método para o tratamento de desrodens neurológicas, pelo aumento da atividade de b-glicocerebrosidase |
| KR101597499B1 (ko) | 2007-05-22 | 2016-02-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 이소파고민과 이의 유도체를 제조하는 새로운 방법 |
-
2007
- 2007-06-25 BR BRPI0713442-8A patent/BRPI0713442A2/pt not_active Application Discontinuation
- 2007-06-25 EP EP20070812295 patent/EP2040548B1/en active Active
- 2007-06-25 JP JP2009518489A patent/JP5303458B2/ja active Active
- 2007-06-25 WO PCT/US2007/072016 patent/WO2007150064A2/en not_active Ceased
- 2007-06-25 DK DK07812295T patent/DK2040548T3/da active
- 2007-06-25 AU AU2007260812A patent/AU2007260812B2/en active Active
- 2007-06-25 ES ES07812295T patent/ES2387845T3/es active Active
- 2007-06-25 KR KR1020097001465A patent/KR101402554B1/ko not_active Expired - Fee Related
- 2007-06-25 CA CA2656643A patent/CA2656643C/en not_active Expired - Fee Related
- 2007-06-25 MX MX2009000032A patent/MX2009000032A/es active IP Right Grant
- 2007-06-25 US US11/768,043 patent/US7829579B2/en active Active
- 2007-06-25 PT PT07812295T patent/PT2040548E/pt unknown
- 2007-06-25 AT AT07812295T patent/ATE555788T1/de active
-
2008
- 2008-12-23 IL IL196151A patent/IL196151A/en active IP Right Grant
-
2009
- 2009-01-13 ZA ZA2009/00270A patent/ZA200900270B/en unknown
-
2010
- 2010-11-08 US US12/941,468 patent/US9119845B2/en active Active
-
2014
- 2014-08-26 US US14/469,008 patent/US9402837B2/en active Active
-
2016
- 2016-08-01 US US15/225,029 patent/US10064851B2/en active Active
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