JP2003528799A5 - - Google Patents
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- JP2003528799A5 JP2003528799A5 JP2000551773A JP2000551773A JP2003528799A5 JP 2003528799 A5 JP2003528799 A5 JP 2003528799A5 JP 2000551773 A JP2000551773 A JP 2000551773A JP 2000551773 A JP2000551773 A JP 2000551773A JP 2003528799 A5 JP2003528799 A5 JP 2003528799A5
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- JP
- Japan
- Prior art keywords
- compound
- calistezin
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- independently represents
- 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.)
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- 150000001875 compounds Chemical class 0.000 description 11
- 238000000034 method Methods 0.000 description 8
- 125000003545 alkoxy group Chemical group 0.000 description 6
- 229910052799 carbon Inorganic materials 0.000 description 6
- 125000002768 hydroxyalkyl group Chemical group 0.000 description 6
- 102000004190 Enzymes Human genes 0.000 description 5
- 108090000790 Enzymes Proteins 0.000 description 5
- 230000002132 lysosomal effect Effects 0.000 description 5
- 208000015439 Lysosomal storage disease Diseases 0.000 description 4
- 239000003814 drug Substances 0.000 description 4
- 238000009825 accumulation Methods 0.000 description 3
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 description 3
- 229950007469 migalastat Drugs 0.000 description 3
- 150000002772 monosaccharides Chemical class 0.000 description 3
- 150000002812 neutral glycosphingolipids Chemical class 0.000 description 3
- 210000003556 vascular endothelial cell Anatomy 0.000 description 3
- GNFHYSIKCKEHAV-NYYRGLJWSA-N (2S,3R,4S,5R,6R)-2-[(2R,3S,4S,5R,6R)-4,5-dihydroxy-2,6-bis(hydroxymethyl)piperidin-3-yl]oxy-6-(hydroxymethyl)oxane-3,4,5-triol Chemical compound O[C@H]1[C@H](O)[C@@H](CO)N[C@H](CO)[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 GNFHYSIKCKEHAV-NYYRGLJWSA-N 0.000 description 2
- 0 C*([C@@]1C[C@@]2C)C(*)(**)C1C2(*)N Chemical compound C*([C@@]1C[C@@]2C)C(*)(**)C1C2(*)N 0.000 description 2
- 208000024720 Fabry Disease Diseases 0.000 description 2
- 102000005840 alpha-Galactosidase Human genes 0.000 description 2
- 108010030291 alpha-Galactosidase Proteins 0.000 description 2
- 238000004519 manufacturing process Methods 0.000 description 2
- PFYHYHZGDNWFIF-UHFFFAOYSA-N (+)-DMDP Natural products OCC1NC(CO)C(O)C1O PFYHYHZGDNWFIF-UHFFFAOYSA-N 0.000 description 1
- PFYHYHZGDNWFIF-KVTDHHQDSA-N 2,5-bis(hydroxymethyl)-3,4-dihydroxypyrrolidine Chemical compound OC[C@H]1N[C@H](CO)[C@@H](O)[C@@H]1O PFYHYHZGDNWFIF-KVTDHHQDSA-N 0.000 description 1
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 description 1
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 229940106189 ceramide Drugs 0.000 description 1
- ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 210000002472 endoplasmic reticulum Anatomy 0.000 description 1
- 150000002339 glycosphingolipids Chemical class 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 208000010125 myocardial infarction Diseases 0.000 description 1
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 description 1
- 230000002028 premature Effects 0.000 description 1
- 230000008085 renal dysfunction Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US09/087,804 US6274597B1 (en) | 1998-06-01 | 1998-06-01 | Method of enhancing lysosomal α-Galactosidase A |
| US09/087,804 | 1998-06-01 | ||
| PCT/US1999/010479 WO1999062517A1 (en) | 1998-06-01 | 1999-05-28 | METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010173733A Division JP5528941B2 (ja) | 1998-06-01 | 2010-08-02 | リソソームα−ガラクトシダーゼＡの増強方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2003528799A JP2003528799A (ja) | 2003-09-30 |
| JP2003528799A5 true JP2003528799A5 (enExample) | 2006-07-13 |
| JP4602548B2 JP4602548B2 (ja) | 2010-12-22 |
Family
ID=22207362
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2000551773A Expired - Lifetime JP4602548B2 (ja) | 1998-06-01 | 1999-05-28 | リソソームα−ガラクトシダーゼＡの増強方法 |
| JP2010173733A Expired - Lifetime JP5528941B2 (ja) | 1998-06-01 | 2010-08-02 | リソソームα−ガラクトシダーゼＡの増強方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010173733A Expired - Lifetime JP5528941B2 (ja) | 1998-06-01 | 2010-08-02 | リソソームα−ガラクトシダーゼＡの増強方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (16) | US6274597B1 (enExample) |
| EP (2) | EP2143420B1 (enExample) |
| JP (2) | JP4602548B2 (enExample) |
| AT (1) | ATE442851T1 (enExample) |
| AU (1) | AU4186199A (enExample) |
| CA (1) | CA2333965C (enExample) |
| CY (2) | CY1112845T1 (enExample) |
| DE (1) | DE69941429D1 (enExample) |
| DK (2) | DK1083899T3 (enExample) |
| ES (2) | ES2507515T3 (enExample) |
| PT (2) | PT2143420E (enExample) |
| WO (1) | WO1999062517A1 (enExample) |
Families Citing this family (105)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6274597B1 (en) * | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
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| CA2398995C (en) | 2000-02-04 | 2014-09-23 | Children's Hospital Research Foundation | Lipid hydrolysis therapy for atherosclerosis and related diseases |
| WO2003022797A1 (en) * | 2001-09-07 | 2003-03-20 | Seikagaku Corporation | Carba-sugar amine derivatives and treatments for disorder of glycolipid metabolism containing the same as the active ingredient |
| US7842470B2 (en) * | 2001-10-09 | 2010-11-30 | Oregon Health & Science University | Method for pharmacoperones correction of GnRHR mutant protein misfolding |
| AU2003281195A1 (en) * | 2002-07-05 | 2004-01-23 | Roderick Frederick Gerardus Joseph King | Aqueous formulation comprising digestible saccharides to reduce the tendency to cardiovascular disease in a subject |
| WO2004037373A2 (en) * | 2002-10-21 | 2004-05-06 | The Scripps Research Institute | CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE |
| PT2444102E (pt) * | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| EP1633356A1 (en) * | 2003-05-22 | 2006-03-15 | The Hospital for Sick Children | Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity |
| FR2861991B1 (fr) | 2003-11-07 | 2008-01-18 | Centre Nat Rech Scient | Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose |
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| FR2873377B1 (fr) * | 2004-07-23 | 2006-10-13 | Sanofi Synthelabo | Derives d'azasucre, inhibiteurs d'heparanases, leur procede de preparation, les compositions en contenant et leur utilisation |
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1998
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1999
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- 1999-05-28 ES ES09011761.5T patent/ES2507515T3/es not_active Expired - Lifetime
- 1999-05-28 ES ES99925611T patent/ES2333561T3/es not_active Expired - Lifetime
- 1999-05-28 DK DK99925611.8T patent/DK1083899T3/da active
- 1999-05-28 WO PCT/US1999/010479 patent/WO1999062517A1/en not_active Ceased
- 1999-05-28 DK DK09011761.5T patent/DK2143420T3/da active
- 1999-05-28 JP JP2000551773A patent/JP4602548B2/ja not_active Expired - Lifetime
- 1999-05-28 EP EP09011761.5A patent/EP2143420B1/en not_active Expired - Lifetime
- 1999-05-28 PT PT99925611T patent/PT1083899E/pt unknown
- 1999-05-28 DE DE69941429T patent/DE69941429D1/de not_active Expired - Lifetime
- 1999-05-28 AU AU41861/99A patent/AU4186199A/en not_active Abandoned
- 1999-05-28 CA CA2333965A patent/CA2333965C/en not_active Expired - Lifetime
- 1999-05-28 EP EP99925611A patent/EP1083899B1/en not_active Expired - Lifetime
- 1999-05-28 AT AT99925611T patent/ATE442851T1/de active
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2000
- 2000-06-26 US US09/604,053 patent/US6583158B1/en not_active Expired - Lifetime
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2001
- 2001-08-10 US US09/927,285 patent/US6774135B2/en not_active Expired - Lifetime
- 2001-09-07 US US09/948,348 patent/US6599919B2/en not_active Expired - Lifetime
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2002
- 2002-06-14 US US10/172,604 patent/US6589964B2/en not_active Expired - Lifetime
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2004
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2005
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2006
- 2006-09-27 US US11/528,903 patent/US7812033B2/en not_active Expired - Fee Related
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2009
- 2009-11-23 US US12/624,026 patent/US8633221B2/en not_active Expired - Fee Related
- 2009-12-16 CY CY20091101305T patent/CY1112845T1/el unknown
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2010
- 2010-08-02 JP JP2010173733A patent/JP5528941B2/ja not_active Expired - Lifetime
- 2010-09-24 US US12/890,014 patent/US8436018B2/en not_active Expired - Fee Related
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2013
- 2013-04-22 US US13/867,914 patent/US8841322B2/en not_active Expired - Fee Related
- 2013-12-20 US US14/136,418 patent/US9265780B2/en not_active Expired - Fee Related
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2014
- 2014-08-18 US US14/461,827 patent/US20150258129A1/en not_active Abandoned
- 2014-09-24 CY CY20141100776T patent/CY1115583T1/el unknown
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2016
- 2016-01-25 US US15/005,371 patent/US20160346262A1/en not_active Abandoned
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