JP2010525084A5 - - Google Patents
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- Publication number
- JP2010525084A5 JP2010525084A5 JP2010506557A JP2010506557A JP2010525084A5 JP 2010525084 A5 JP2010525084 A5 JP 2010525084A5 JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010525084 A5 JP2010525084 A5 JP 2010525084A5
- Authority
- JP
- Japan
- Prior art keywords
- disease
- pharmacological chaperone
- use according
- days
- maintenance dose
- 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.)
- Pending
Links
- 230000000144 pharmacologic effect Effects 0.000 claims 8
- 201000010099 disease Diseases 0.000 claims 7
- 201000005603 Fabry disease Diseases 0.000 claims 4
- LXBIFEVIBLOUGU-DPYQTVNSSA-N (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol Chemical group OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 claims 3
- 229950007469 migalastat Drugs 0.000 claims 3
- 102100008255 GAA Human genes 0.000 claims 2
- 101710010383 GAA Proteins 0.000 claims 2
- 206010018048 Gaucher's disease Diseases 0.000 claims 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 2
- 201000004502 glycogen storage disease II Diseases 0.000 claims 2
- LXBIFEVIBLOUGU-FSIIMWSLSA-N 1,5-Dideoxy-1,5-Imino-D-Mannitol Chemical group OC[C@@H]1NC[C@@H](O)[C@H](O)[C@H]1O LXBIFEVIBLOUGU-FSIIMWSLSA-N 0.000 claims 1
- LXBIFEVIBLOUGU-JGWLITMVSA-N 1-Deoxynojirimycin Natural products OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O LXBIFEVIBLOUGU-JGWLITMVSA-N 0.000 claims 1
- QPYJXFZUIJOGNX-HSUXUTPPSA-N Afegostat Chemical group OC[C@H]1CNC[C@@H](O)[C@@H]1O QPYJXFZUIJOGNX-HSUXUTPPSA-N 0.000 claims 1
- 229940022705 Aldurazyme Drugs 0.000 claims 1
- 206010001897 Alzheimer's disease Diseases 0.000 claims 1
- 229940049197 Cerezyme Drugs 0.000 claims 1
- 229940014516 Fabrazyme Drugs 0.000 claims 1
- UQRORFVVSGFNRO-UTINFBMNSA-N Miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims 1
- 229960001512 Miglustat Drugs 0.000 claims 1
- 229940103023 Myozyme Drugs 0.000 claims 1
- 206010061536 Parkinson's disease Diseases 0.000 claims 1
- 229940099072 Zavesca Drugs 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000011780 sodium chloride Substances 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Claims (9)
- タンパク質の誤った折畳みに関連する疾病の治療に使用するための薬理シャペロンであって、ここに治療が約４日〜約１０日間１日１回薬理シャペロン約７５〜３００ｍｇの日用量を経口投与し、続いて約３〜約８日おきに１回約７５〜２２５ｍｇの維持用量を経口投与することを含む、薬理シャペロン。
- 投与される維持用量が約１５０ｍｇである、請求項１に記載の使用。
- 維持用量が約２〜３日おきに１回投与され、薬理シャペロンが１−デオキシガラクトノジリマイシンまたはその薬理学的に許容される塩であって、疾病がファブリー病である、請求項１に記載の使用。
- 疾病がファブリー病、ゴーシェ病、ポンペ病、パーキンソン病、およびアルツハイマ病からなる群から選択される、請求項１に記載の使用。
- 薬理シャペロンが１−デオキシガラクトノジリマイシンであって、疾病がファブリー病である、請求項１に記載の使用。
- 疾病がファブリー病であり、薬理シャペロンが１−デオキシガラクトノジリマイシンであって、維持用量が２〜３日おきに約１５０ｍｇである、請求項１に記載の使用。
- Ｃｅｒｅｚｙｍｅ（登録商標）、Ｆａｂｒａｚｙｍｅ（登録商標）、Ａｌｄｕｒａｚｙｍｅ（登録商標）、Ｍｙｏｚｙｍｅ（登録商標）、Ｒｅｐｌａｇａｌ（登録商標）およびＺａｖｅｓｃａ（登録商標）からなる群から選択される少なくとも１つの追加療法と組合せて薬理シャペロンを投与するステップをさらに含む、請求項１に記載の使用。
- 薬理シャペロンがイソファゴミンであって、疾病がゴーシェ病である、請求項１に記載の使用。
- 薬理シャペロンが１−デオキシノジリマイシンであって、疾病がポンペ病である、請求項１に記載の使用。
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US91428807P | 2007-04-26 | 2007-04-26 | |
| US1474407P | 2007-12-18 | 2007-12-18 | |
| US2810508P | 2008-02-12 | 2008-02-12 | |
| PCT/US2008/061764 WO2008134628A2 (en) | 2007-04-26 | 2008-04-28 | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2010525084A JP2010525084A (ja) | 2010-07-22 |
| JP2010525084A5 true JP2010525084A5 (ja) | 2011-05-19 |
Family
ID=39926311
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010506557A Pending JP2010525084A (ja) | 2007-04-26 | 2008-04-28 | 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9056101B2 (ja) |
| EP (1) | EP2150254A4 (ja) |
| JP (1) | JP2010525084A (ja) |
| AU (1) | AU2008245578A1 (ja) |
| CA (1) | CA2685332A1 (ja) |
| IL (1) | IL201733A0 (ja) |
| MX (1) | MX2009011473A (ja) |
| WO (1) | WO2008134628A2 (ja) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040180419A1 (en) | 2003-01-31 | 2004-09-16 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| SI2533050T1 (sl) | 2006-05-16 | 2014-07-31 | Amicus Therapeutics, Inc. | MoĹľnosti zdravljenja Fabrijeve bolezni |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| PT2252313E (pt) | 2008-02-12 | 2015-08-26 | Amicus Therapeutics Inc | Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas |
| ES2963910T3 (es) | 2008-06-26 | 2024-04-03 | Zevra Denmark As | Uso de Hsp70 como regulador de la actividad enzimática |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| US20100119502A1 (en) * | 2008-11-11 | 2010-05-13 | Amicus Therapeutics, Inc. | Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease |
| GB0906159D0 (en) * | 2009-04-09 | 2009-05-20 | Summit Corp Plc | Drug combination for the treatment of proteostatic diseases |
| CA2758271C (en) * | 2009-04-09 | 2018-06-05 | Robert Boyd | Methods for preventing and/or treating lysosomal storage disorders |
| ES2549504T3 (es) * | 2009-10-19 | 2015-10-28 | Amicus Therapeutics, Inc. | Método para el tratamiento de la enfermedad de Alzheimer utilizando chaperonas farmacológicas para aumentar la actividad de gangliosidasas |
| CN105193802A (zh) | 2009-10-19 | 2015-12-30 | 阿米库斯治疗学公司 | 用于预防和/或治疗溶酶体贮积失调的新颖组合物 |
| 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 |
| HUE029150T2 (hu) * | 2009-10-27 | 2017-02-28 | Erytech Pharma | Készítmény specifikus immuntolerancia indukálására |
| US9066939B2 (en) * | 2009-11-17 | 2015-06-30 | Baylor Research Institute | Urinary triaosylceramide (GB3) as a marker of cardiac disease |
| SG10201407881WA (en) | 2009-11-27 | 2015-01-29 | Genzyme Corp | An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase |
| CN105296447A (zh) | 2010-11-08 | 2016-02-03 | 阿米库斯治疗学公司 | 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白 |
| EP2646044B1 (en) | 2010-11-30 | 2019-08-28 | Orphazyme A/S | Methods for increasing intracellular activity of hsp70 |
| CN103974619B (zh) * | 2011-03-11 | 2017-02-15 | 阿米库斯治疗学公司 | 治疗法布里病的给药方案 |
| EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
| KR20230066482A (ko) | 2012-03-07 | 2023-05-15 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| EP2831043B1 (en) | 2012-03-27 | 2022-07-27 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
| EP2844279B1 (en) * | 2012-05-03 | 2020-12-09 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of pompe disease |
| US9694056B2 (en) | 2012-07-17 | 2017-07-04 | Amicus Therapeutics, Inc. | α-galactosidase A and 1-deoxygalactonojirimycin co-formulation |
| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| US9675627B2 (en) | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| AU2015317447B2 (en) | 2014-09-15 | 2021-02-25 | Zevra Denmark A/S | Arimoclomol formulation |
| RS65066B1 (sr) | 2014-09-30 | 2024-02-29 | Amicus Therapeutics Inc | Visoko potentna kisela alfa-glukozidaza sa pojačanim ugljenim hidratima |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| US10179128B2 (en) * | 2015-08-31 | 2019-01-15 | Amicus Therapeutics, Inc. | Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system |
| WO2017041051A1 (en) | 2015-09-04 | 2017-03-09 | Sqz Biotechnologies Company | Intracellular delivery of biomolecules to cells comprising a cell wall |
| AU2016381832B2 (en) | 2015-12-30 | 2024-01-25 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of Pompe disease |
| JP6438421B2 (ja) * | 2016-02-17 | 2018-12-12 | ファナック株式会社 | 電動機のステータ |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| KR20240001291A (ko) | 2016-03-30 | 2024-01-03 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| HUE052158T2 (hu) | 2016-04-29 | 2021-04-28 | Orphazyme As | Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| KR20190134807A (ko) | 2017-04-25 | 2019-12-04 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추 신경계의 퇴행성 질환 및/또는 리소솜 저장병을 예방 및/또는 치료하기 위한 신규한 조성물 |
| MX2019014410A (es) | 2017-05-30 | 2020-02-10 | Amicus Therapeutics Inc | Metodos de tratamiento de pacientes con enfermedad de fabry que tienen insuficiencia renal. |
| EP4062915A1 (en) * | 2017-05-30 | 2022-09-28 | Amicus Therapeutics, Inc. | Migalastat bound to alpha galactosidase a |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| KR20220019796A (ko) * | 2019-06-11 | 2022-02-17 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법 |
| AU2020327019A1 (en) | 2019-08-07 | 2022-03-03 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| JP2024500632A (ja) | 2020-11-19 | 2024-01-10 | ゼブラ デンマーク エー／エス | アリモクロモルクエン酸塩及びその中間体の調製プロセス |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (9)
| 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 |
| US20040180419A1 (en) * | 2003-01-31 | 2004-09-16 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US9181184B2 (en) * | 2005-05-17 | 2015-11-10 | Amicus Therapeutics, Inc. | Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| KR20080033242A (ko) | 2005-06-08 | 2008-04-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는ｃｎｓ 질환의 치료 방법 |
| US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
| JP2009502954A (ja) * | 2005-07-27 | 2009-01-29 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド | タンパク質のミスフォールディングを修正する小分子及びその使用 |
| SI2533050T1 (sl) * | 2006-05-16 | 2014-07-31 | Amicus Therapeutics, Inc. | MoĹľnosti zdravljenja Fabrijeve bolezni |
| EP2142197A4 (en) * | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS |
-
2008
- 2008-04-28 US US12/597,238 patent/US9056101B2/en active Active
- 2008-04-28 MX MX2009011473A patent/MX2009011473A/es not_active Application Discontinuation
- 2008-04-28 AU AU2008245578A patent/AU2008245578A1/en not_active Abandoned
- 2008-04-28 EP EP08747020A patent/EP2150254A4/en not_active Withdrawn
- 2008-04-28 WO PCT/US2008/061764 patent/WO2008134628A2/en active Application Filing
- 2008-04-28 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
- 2008-04-28 JP JP2010506557A patent/JP2010525084A/ja active Pending
-
2009
- 2009-10-25 IL IL201733A patent/IL201733A0/en unknown
-
2015
- 2015-05-15 US US14/713,821 patent/US20150352093A1/en not_active Abandoned
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