JP2002531581A5 - - Google Patents
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- Publication number
- JP2002531581A5 JP2002531581A5 JP2000586885A JP2000586885A JP2002531581A5 JP 2002531581 A5 JP2002531581 A5 JP 2002531581A5 JP 2000586885 A JP2000586885 A JP 2000586885A JP 2000586885 A JP2000586885 A JP 2000586885A JP 2002531581 A5 JP2002531581 A5 JP 2002531581A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- dosage
- composition according
- glucosidase
- patient
- 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
- 239000008194 pharmaceutical composition Substances 0.000 description 36
- 239000000203 mixture Substances 0.000 description 11
- 102000016679 alpha-Glucosidases Human genes 0.000 description 8
- 108010028144 alpha-Glucosidases Proteins 0.000 description 8
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 description 7
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 description 7
- 229930195725 Mannitol Natural products 0.000 description 7
- 229930006000 Sucrose Natural products 0.000 description 7
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 7
- 102000045921 human GAA Human genes 0.000 description 7
- 239000000594 mannitol Substances 0.000 description 7
- 235000010355 mannitol Nutrition 0.000 description 7
- 239000005720 sucrose Substances 0.000 description 7
- 239000007864 aqueous solution Substances 0.000 description 5
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 3
- 230000037396 body weight Effects 0.000 description 3
- 201000004502 glycogen storage disease II Diseases 0.000 description 3
- 102000008100 Human Serum Albumin Human genes 0.000 description 2
- 108091006905 Human Serum Albumin Proteins 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 238000000034 method Methods 0.000 description 2
- 239000011780 sodium chloride Substances 0.000 description 2
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
- 102000004366 Glucosidases Human genes 0.000 description 1
- 108010056771 Glucosidases Proteins 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 239000008103 glucose Substances 0.000 description 1
- 230000000366 juvenile effect Effects 0.000 description 1
- 235000013336 milk Nutrition 0.000 description 1
- 239000008267 milk Substances 0.000 description 1
- 210000004080 milk Anatomy 0.000 description 1
- 238000012544 monitoring process Methods 0.000 description 1
- 239000012064 sodium phosphate buffer Substances 0.000 description 1
- 230000009261 transgenic effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11129198P | 1998-12-07 | 1998-12-07 | |
| US60/111,291 | 1998-12-07 | ||
| PCT/US1999/029042 WO2000034451A1 (en) | 1998-12-07 | 1999-12-06 | Treatment of pompe's disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009102515A Division JP2009161562A (ja) | 1998-12-07 | 2009-04-20 | ポンペ病の処置 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2002531581A JP2002531581A (ja) | 2002-09-24 |
| JP2002531581A5 true JP2002531581A5 (enExample) | 2006-02-02 |
| JP4990434B2 JP4990434B2 (ja) | 2012-08-01 |
Family
ID=22337648
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2000586885A Expired - Lifetime JP4990434B2 (ja) | 1998-12-07 | 1999-12-06 | ポンペ病の処置 |
| JP2009102515A Withdrawn JP2009161562A (ja) | 1998-12-07 | 2009-04-20 | ポンペ病の処置 |
| JP2012181032A Withdrawn JP2012224643A (ja) | 1998-12-07 | 2012-08-17 | ポンペ病の処置 |
| JP2015094511A Withdrawn JP2015134836A (ja) | 1998-12-07 | 2015-05-05 | ポンペ病の処置 |
| JP2017000155A Withdrawn JP2017066160A (ja) | 1998-12-07 | 2017-01-04 | ポンペ病の処置 |
| JP2018061663A Withdrawn JP2018115200A (ja) | 1998-12-07 | 2018-03-28 | ポンペ病の処置 |
| JP2019105319A Pending JP2019172685A (ja) | 1998-12-07 | 2019-06-05 | ポンペ病の処置 |
Family Applications After (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009102515A Withdrawn JP2009161562A (ja) | 1998-12-07 | 2009-04-20 | ポンペ病の処置 |
| JP2012181032A Withdrawn JP2012224643A (ja) | 1998-12-07 | 2012-08-17 | ポンペ病の処置 |
| JP2015094511A Withdrawn JP2015134836A (ja) | 1998-12-07 | 2015-05-05 | ポンペ病の処置 |
| JP2017000155A Withdrawn JP2017066160A (ja) | 1998-12-07 | 2017-01-04 | ポンペ病の処置 |
| JP2018061663A Withdrawn JP2018115200A (ja) | 1998-12-07 | 2018-03-28 | ポンペ病の処置 |
| JP2019105319A Pending JP2019172685A (ja) | 1998-12-07 | 2019-06-05 | ポンペ病の処置 |
Country Status (16)
| Country | Link |
|---|---|
| US (7) | US20030007963A1 (enExample) |
| EP (2) | EP2020438B1 (enExample) |
| JP (7) | JP4990434B2 (enExample) |
| KR (1) | KR20010101131A (enExample) |
| AT (1) | ATE410506T1 (enExample) |
| AU (1) | AU3113700A (enExample) |
| BE (1) | BE2018C038I2 (enExample) |
| CA (1) | CA2353522A1 (enExample) |
| CY (2) | CY1121113T1 (enExample) |
| DE (2) | DE69939705D1 (enExample) |
| DK (2) | DK2020438T3 (enExample) |
| ES (2) | ES2312222T3 (enExample) |
| LU (1) | LU91544I2 (enExample) |
| NL (1) | NL300382I2 (enExample) |
| PT (2) | PT1137762E (enExample) |
| WO (1) | WO2000034451A1 (enExample) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU3113700A (en) * | 1998-12-07 | 2000-06-26 | Genzyme Corporation | Treatment of pompe's disease |
| EP1267914B2 (en) * | 2000-02-04 | 2013-11-06 | Children's Hospital Research Foundation | Use of lysosomal acid lipase for treating atherosclerosis and related diseases |
| EP1782825B1 (en) * | 2000-07-18 | 2014-09-10 | Duke University | Treatment of glycogen storage diseases type II |
| AU2001271941B2 (en) * | 2000-07-18 | 2007-03-01 | Duke University | Treatment of glycogen storage disease type II |
| AU2016256663A1 (en) * | 2000-07-18 | 2016-11-24 | Duke University | Treatment of clycogen storage disease type II |
| US7723296B2 (en) * | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| ES2300439T3 (es) * | 2001-04-30 | 2008-06-16 | Zystor Therapeutics , Inc. | Reconocimiento subcelular de proteinas terapeuticas. |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| WO2003086452A2 (en) | 2002-04-05 | 2003-10-23 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy |
| WO2004064750A2 (en) | 2003-01-22 | 2004-08-05 | Duke University | Improved constructs for expressing lysosomal polypeptides |
| WO2004069190A2 (en) | 2003-01-31 | 2004-08-19 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| CN1922313B (zh) * | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| KR100697270B1 (ko) * | 2004-12-10 | 2007-03-21 | 삼성전자주식회사 | 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법 |
| EP3441090A1 (en) | 2005-05-17 | 2019-02-13 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
| WO2008063511A2 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Methods for treating pompe disease |
| HRP20170698T1 (hr) | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| HUE025212T2 (en) * | 2008-07-08 | 2016-03-29 | Univ Duke | A method for treating glycogen storage disease |
| EP2475376B1 (en) | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| US8536148B2 (en) | 2009-09-04 | 2013-09-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Disabling autophagy as a treatment for lysosomal storage diseases |
| AU2011242461B2 (en) | 2010-04-23 | 2015-10-01 | Alexion Pharmaceuticals, Inc. | Lysosomal storage disease enzyme |
| SMT201600385T1 (it) | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| KR102720061B1 (ko) | 2010-06-25 | 2024-10-22 | 다케다 파머수티컬 컴패니 리미티드 | 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 |
| MX2013000324A (es) | 2010-06-25 | 2013-02-01 | Shire Human Genetic Therapies | Tratamiento del sindrome de sanfilippo tipo b. |
| UA115649C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Способи та композиції для доставки до цнс ідуронат-2-сульфатази |
| BR112012033197A2 (pt) | 2010-06-25 | 2017-06-06 | Shire Human Genetic Therapies | métodos e composições para liberação de cns de n-sulfatase de heparano. |
| PT2588130T (pt) | 2010-06-25 | 2016-11-25 | Shire Human Genetic Therapies | Entrega de agentes terapêuticos no cns |
| AR082953A1 (es) | 2010-09-09 | 2013-01-23 | Synageva Biopharma Corp | Metodos para tratar deficiencia de acido lisosomal lipasa en pacientes |
| KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
| DK2687597T3 (en) | 2011-03-16 | 2019-02-25 | Amano Enzyme Inc | MODIFIED ALPHA-GLUCOSIDASE AND USES OF SAME |
| BR112013026976A2 (pt) * | 2011-04-22 | 2019-09-24 | Genzyme Corp | alfa glucosidade ácida modificada com processamento acelerado |
| EP2793922B1 (en) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| KR20230066482A (ko) | 2012-03-07 | 2023-05-15 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| HK1220980A1 (zh) | 2013-02-20 | 2017-05-19 | Valerion Therapeutics, Llc | 用於治疗庞贝氏症的方法和组合物 |
| US10603364B2 (en) | 2014-08-11 | 2020-03-31 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| MY186336A (en) | 2014-09-30 | 2021-07-13 | Amicus Therapeutics Inc | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
| US20180326021A1 (en) * | 2015-08-31 | 2018-11-15 | Duke University | Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders |
| US10940125B2 (en) | 2015-09-18 | 2021-03-09 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
| CA3010205A1 (en) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| MX2018011951A (es) | 2016-03-30 | 2019-02-13 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p. |
| EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| IL319481A (en) | 2016-09-12 | 2025-05-01 | Inst Nat Sante Rech Med | Variants of acid alpha-glucosidase and their uses |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| EP3996696A1 (en) | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| JP7689746B2 (ja) * | 2019-12-17 | 2025-06-09 | ナフィゴ プロテインズ ゲゼルシャフト ミット ベシュレンクテル ハフツング | 酵素酸性αグルコシダーゼ（ＧＡＡ）の結合タンパク質及びその使用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0832981A1 (en) | 1987-02-17 | 1998-04-01 | Pharming B.V. | DNA sequences to target proteins to the mammary gland for efficient secretion |
| EP0502976B1 (en) | 1989-12-01 | 1996-07-03 | Pharming B.V. | Production of recombinant polypeptides by bovine species and transgenic methods |
| AU664976B2 (en) | 1990-08-29 | 1995-12-14 | Gene Pharming Europe Bv | Homologous recombination in mammalian cells |
| US5356804A (en) | 1990-10-24 | 1994-10-18 | Mount Sinai School Of Medicine Of The City Of New York | Cloning and expression of biologically active human α-galactosidase A |
| AU4537393A (en) | 1992-06-15 | 1994-01-04 | Gene Pharming Europe Bv | Production of recombinant polypeptides by bovine species and transgenic methods |
| CA2123671C (en) * | 1993-05-17 | 1999-08-03 | Mitsuo Yamada | Oxazolidone ring-containing modified epoxy resins and cathodic electrodeposition paints containing same |
| AT1470U1 (de) | 1995-08-01 | 1997-05-26 | Austria Card | Laminierte karte und verfahren zu ihrer herstellung |
| US6118045A (en) * | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| AU3113700A (en) * | 1998-12-07 | 2000-06-26 | Genzyme Corporation | Treatment of pompe's disease |
-
1999
- 1999-12-06 AU AU31137/00A patent/AU3113700A/en not_active Abandoned
- 1999-12-06 ES ES99965162T patent/ES2312222T3/es not_active Expired - Lifetime
- 1999-12-06 JP JP2000586885A patent/JP4990434B2/ja not_active Expired - Lifetime
- 1999-12-06 DE DE69939705T patent/DE69939705D1/de not_active Expired - Lifetime
- 1999-12-06 EP EP08164825.5A patent/EP2020438B1/en not_active Revoked
- 1999-12-06 ES ES08164825.5T patent/ES2677343T3/es not_active Expired - Lifetime
- 1999-12-06 US US09/454,711 patent/US20030007963A1/en not_active Abandoned
- 1999-12-06 DK DK08164825.5T patent/DK2020438T3/en active
- 1999-12-06 PT PT99965162T patent/PT1137762E/pt unknown
- 1999-12-06 EP EP99965162A patent/EP1137762B1/en not_active Revoked
- 1999-12-06 CA CA002353522A patent/CA2353522A1/en not_active Abandoned
- 1999-12-06 WO PCT/US1999/029042 patent/WO2000034451A1/en not_active Ceased
- 1999-12-06 DE DE122009000018C patent/DE122009000018I1/de active Pending
- 1999-12-06 DK DK99965162T patent/DK1137762T3/da active
- 1999-12-06 AT AT99965162T patent/ATE410506T1/de active
- 1999-12-06 PT PT81648255T patent/PT2020438T/pt unknown
- 1999-12-06 KR KR1020017007040A patent/KR20010101131A/ko not_active Withdrawn
-
2003
- 2003-06-30 US US10/611,598 patent/US7351410B2/en not_active Expired - Lifetime
-
2008
- 2008-01-30 US US12/012,003 patent/US7655226B2/en not_active Expired - Fee Related
-
2009
- 2009-03-24 NL NL300382C patent/NL300382I2/nl unknown
- 2009-03-25 LU LU91544C patent/LU91544I2/fr unknown
- 2009-04-20 JP JP2009102515A patent/JP2009161562A/ja not_active Withdrawn
- 2009-12-09 US US12/633,932 patent/US20100092449A1/en not_active Abandoned
-
2011
- 2011-12-21 US US13/333,052 patent/US20130039901A1/en not_active Abandoned
-
2012
- 2012-08-17 JP JP2012181032A patent/JP2012224643A/ja not_active Withdrawn
-
2013
- 2013-05-03 US US13/886,573 patent/US20140037611A1/en not_active Abandoned
-
2015
- 2015-05-05 JP JP2015094511A patent/JP2015134836A/ja not_active Withdrawn
- 2015-09-23 US US14/863,333 patent/US20160243203A1/en not_active Abandoned
-
2017
- 2017-01-04 JP JP2017000155A patent/JP2017066160A/ja not_active Withdrawn
-
2018
- 2018-03-28 JP JP2018061663A patent/JP2018115200A/ja not_active Withdrawn
- 2018-07-10 CY CY20181100718T patent/CY1121113T1/el unknown
- 2018-07-25 CY CY2018020C patent/CY2018020I2/el unknown
- 2018-09-28 BE BE2018C038C patent/BE2018C038I2/fr unknown
-
2019
- 2019-06-05 JP JP2019105319A patent/JP2019172685A/ja active Pending
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