JP2009137933A5 - - Google Patents
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- Publication number
- JP2009137933A5 JP2009137933A5 JP2008135368A JP2008135368A JP2009137933A5 JP 2009137933 A5 JP2009137933 A5 JP 2009137933A5 JP 2008135368 A JP2008135368 A JP 2008135368A JP 2008135368 A JP2008135368 A JP 2008135368A JP 2009137933 A5 JP2009137933 A5 JP 2009137933A5
- Authority
- JP
- Japan
- Prior art keywords
- gcase
- ambroxol
- compound
- shows
- abx
- 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
- JBDGDEWWOUBZPM-XYPYZODXSA-N ambroxol Chemical compound NC1=C(Br)C=C(Br)C=C1CN[C@@H]1CC[C@@H](O)CC1 JBDGDEWWOUBZPM-XYPYZODXSA-N 0.000 claims description 15
- 229960005174 ambroxol Drugs 0.000 claims description 15
- 208000015872 Gaucher disease Diseases 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- 229940079593 drug Drugs 0.000 claims description 5
- 150000001875 compounds Chemical class 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 3
- BQIMTENFTIAWGJ-UHFFFAOYSA-N (2,3,4-tribromophenyl) propanoate Chemical compound CCC(=O)OC1=CC=C(Br)C(Br)=C1Br BQIMTENFTIAWGJ-UHFFFAOYSA-N 0.000 claims 1
- GVPODVKBTHCGFU-UHFFFAOYSA-N 2,4,6-tribromoaniline Chemical compound NC1=C(Br)C=C(Br)C=C1Br GVPODVKBTHCGFU-UHFFFAOYSA-N 0.000 claims 1
- QNVKOSLOVOTXKF-UHFFFAOYSA-N 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol;hydron;chloride Chemical compound Cl.NC1=C(Br)C=C(Br)C=C1CNC1CCC(O)CC1 QNVKOSLOVOTXKF-UHFFFAOYSA-N 0.000 claims 1
- HFEHQMJHVHTNJN-UHFFFAOYSA-N BrC1=C(C(=C(C(=O)N)C=C1)C1=CC=CC=C1)Br Chemical compound BrC1=C(C(=C(C(=O)N)C=C1)C1=CC=CC=C1)Br HFEHQMJHVHTNJN-UHFFFAOYSA-N 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 229960000985 ambroxol hydrochloride Drugs 0.000 claims 1
- -1 bromohexine Chemical compound 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- ZDHOYZBANMCKGV-UHFFFAOYSA-N n,n-dibromoaniline Chemical compound BrN(Br)C1=CC=CC=C1 ZDHOYZBANMCKGV-UHFFFAOYSA-N 0.000 claims 1
- 230000000694 effects Effects 0.000 description 8
- 102000004169 proteins and genes Human genes 0.000 description 5
- 108090000623 proteins and genes Proteins 0.000 description 5
- 230000032258 transport Effects 0.000 description 5
- 210000002950 fibroblast Anatomy 0.000 description 4
- 238000010186 staining Methods 0.000 description 4
- 101150048357 Lamp1 gene Proteins 0.000 description 3
- QPYJXFZUIJOGNX-HSUXUTPPSA-N afegostat Chemical compound OC[C@H]1CNC[C@@H](O)[C@@H]1O QPYJXFZUIJOGNX-HSUXUTPPSA-N 0.000 description 3
- 230000008045 co-localization Effects 0.000 description 3
- 230000004927 fusion Effects 0.000 description 3
- 210000003712 lysosome Anatomy 0.000 description 3
- 230000001868 lysosomic effect Effects 0.000 description 3
- 102000014944 Lysosome-Associated Membrane Glycoproteins Human genes 0.000 description 2
- 108010064171 Lysosome-Associated Membrane Glycoproteins Proteins 0.000 description 2
- 241000283973 Oryctolagus cuniculus Species 0.000 description 2
- 210000004027 cell Anatomy 0.000 description 2
- 238000003271 compound fluorescence assay Methods 0.000 description 2
- 230000035772 mutation Effects 0.000 description 2
- 238000012758 nuclear staining Methods 0.000 description 2
- 208000020322 Gaucher disease type I Diseases 0.000 description 1
- 108010006519 Molecular Chaperones Proteins 0.000 description 1
- 230000002378 acidificating effect Effects 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 229940049197 cerezyme Drugs 0.000 description 1
- 230000007423 decrease Effects 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 238000012757 fluorescence staining Methods 0.000 description 1
- 238000003505 heat denaturation Methods 0.000 description 1
- 108010039650 imiglucerase Proteins 0.000 description 1
- 230000002055 immunohistochemical effect Effects 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 230000000144 pharmacologic effect Effects 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US97296807P | 2007-09-17 | 2007-09-17 | |
| US60/972,968 | 2007-09-17 | ||
| US6555008P | 2008-02-12 | 2008-02-12 | |
| US61/065,550 | 2008-02-12 | ||
| US6568408P | 2008-02-13 | 2008-02-13 | |
| US61/065,684 | 2008-02-13 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2009137933A JP2009137933A (ja) | 2009-06-25 |
| JP2009137933A5 true JP2009137933A5 (https=) | 2011-06-16 |
| JP5337405B2 JP5337405B2 (ja) | 2013-11-06 |
Family
ID=39875224
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008135368A Active JP5337405B2 (ja) | 2007-09-17 | 2008-05-23 | ゴーシェ病の治療方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (10) | US8124597B2 (https=) |
| EP (1) | EP2200596B1 (https=) |
| JP (1) | JP5337405B2 (https=) |
| CA (1) | CA2699864C (https=) |
| IL (1) | IL204285A (https=) |
| WO (1) | WO2009038695A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE102004021992A1 (de) * | 2004-05-03 | 2005-11-24 | Boehringer Ingelheim Pharma Gmbh & Co. Kg | Topische Zubereitung enthaltend Ambroxol |
| EP3366688B1 (en) | 2010-12-08 | 2022-02-09 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Substituted pyrazolopyrimidines as glucocerebrosidase activators |
| KR102076585B1 (ko) * | 2011-12-22 | 2020-02-12 | 센토제너 아이피 게엠베하 | 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 |
| KR102227473B1 (ko) | 2013-04-15 | 2021-03-15 | 삼성디스플레이 주식회사 | 박막 증착 장치 및 이를 이용한 박막 형성 방법 |
| CN106455661A (zh) * | 2014-05-21 | 2017-02-22 | 雀巢产品技术援助有限公司 | 营养物质的个性化补充 |
| US20190254992A1 (en) * | 2016-06-29 | 2019-08-22 | Hadasit Medical Research Services & Development Limited | Combinations of beta-glycolipides and 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol, compositions and uses thereof in the treatment of disorders associated with protein misfolding and protein aggregations |
| WO2018089797A1 (en) | 2016-11-14 | 2018-05-17 | Mingwu Wang | Formulations for the treatment of ocular surface diseases and related methods |
| CN110325516A (zh) | 2016-12-29 | 2019-10-11 | 米尼奥尔克斯治疗有限公司 | 杂芳基化合物和它们的用途 |
| JP7369716B2 (ja) * | 2018-01-26 | 2023-10-26 | ノイレ， エルエルシー | 皮膚バリア機能を改善するためのアンブロキソールの使用 |
| WO2019211205A1 (en) | 2018-04-30 | 2019-11-07 | Dorphan S.A. | Pharmaceutical compositions for use in the treatment of lysosomal diseases |
| 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 |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| KR20210148101A (ko) | 2019-02-04 | 2021-12-07 | 프리라인 테라퓨틱스 리미티드 | 폴리뉴클레오티드 |
| WO2025153714A1 (en) * | 2024-01-19 | 2025-07-24 | Scenic Biotech Bv | Pla2g15 inhibitors for use in the treatment of lysosomal storage diseases, hiv, alzheimer's disease or parkinson's disease |
| CN118086341B (zh) * | 2024-04-25 | 2024-08-02 | 上海凌医生物科技有限公司 | 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ID28460A (id) | 1998-07-08 | 2001-05-24 | Lipogenics Inc | Komposisi dan metoda untuk perlakuan dan pencegahan penyakit tulang dengan menggunakan tocotrienol |
| MXPA04002066A (es) * | 2001-09-04 | 2005-02-17 | Techno Network Shinoku Co Ltd | Farmacos contra influenza. |
| US20030171391A1 (en) * | 2002-01-25 | 2003-09-11 | Boehringer Ingelheim Pharma Gmbh & Co. Kg | Ambroxol for the treatment of chronic pain |
| DE10303229B4 (de) * | 2003-01-28 | 2007-07-26 | Keyneurotek Ag | Ambroxol und Inhibitoren des Angiotensin Converting-Enzyms (ACE) umfassende Arzneimittel Arzneimittel-Zubereitung und ihre Verwendung zur Behandlung von neurodegenerativen Erkrankungen |
| DE10332472A1 (de) * | 2003-07-16 | 2005-02-03 | Boehringer Ingelheim Pharma Gmbh & Co. Kg | Ambroxol für die Behandlung von Tinnitus |
| US7488721B2 (en) * | 2004-05-13 | 2009-02-10 | Mcmaster University | Real time methylumbelliferone-based assay |
| EP1850840A2 (en) * | 2005-01-13 | 2007-11-07 | Sirtris Pharmaceuticals, Inc. | Novel compositions for preventing and treating neurodegenerative and blood coagulation disorders |
| JP2008528510A (ja) * | 2005-01-20 | 2008-07-31 | サートリス ファーマシューティカルズ， インコーポレイテッド | 紅潮および／または薬物誘発性体重増加を処置するためのサーチュイン活性化化合物の使用 |
| US20070149466A1 (en) * | 2005-07-07 | 2007-06-28 | Michael Milburn | Methods and related compositions for treating or preventing obesity, insulin resistance disorders, and mitochondrial-associated disorders |
| US8795668B2 (en) * | 2005-12-23 | 2014-08-05 | The Regents Of The University Of Michigan | Methods for treating pulmonary fibrosis |
| US20070244184A1 (en) * | 2006-01-09 | 2007-10-18 | Simon Fraser University | Glycosidase inhibitors and methods of synthesizing same |
-
2008
- 2008-05-23 JP JP2008135368A patent/JP5337405B2/ja active Active
- 2008-08-22 US US12/229,445 patent/US8124597B2/en not_active Expired - Fee Related
- 2008-09-16 WO PCT/US2008/010768 patent/WO2009038695A1/en not_active Ceased
- 2008-09-16 CA CA2699864A patent/CA2699864C/en active Active
- 2008-09-16 EP EP08831884A patent/EP2200596B1/en active Active
-
2010
- 2010-03-03 IL IL204285A patent/IL204285A/en active IP Right Grant
-
2011
- 2011-09-29 US US13/248,434 patent/US8404668B2/en active Active
-
2012
- 2012-11-14 US US13/676,506 patent/US8951994B2/en active Active
-
2013
- 2013-02-25 US US13/775,388 patent/US9006223B2/en active Active
-
2014
- 2014-04-17 US US14/255,479 patent/US8937059B2/en active Active
- 2014-04-17 US US14/255,324 patent/US9233083B2/en active Active
-
2015
- 2015-12-21 US US14/977,251 patent/US9415025B2/en active Active
-
2016
- 2016-07-29 US US15/223,312 patent/US10028922B2/en active Active
-
2018
- 2018-06-21 US US16/014,739 patent/US10653645B2/en active Active
-
2020
- 2020-04-20 US US16/853,579 patent/US11160773B2/en active Active
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