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
- 108010006519 Molecular Chaperones Proteins 0.000 claims 8
- 230000000144 pharmacologic effect Effects 0.000 claims 8
- 201000010099 disease Diseases 0.000 claims 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 7
- 208000024720 Fabry Disease Diseases 0.000 claims 4
- 238000012423 maintenance Methods 0.000 claims 4
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat 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
- 208000015872 Gaucher disease Diseases 0.000 claims 2
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 2
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- LXBIFEVIBLOUGU-JGWLITMVSA-N duvoglustat Chemical group OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O LXBIFEVIBLOUGU-JGWLITMVSA-N 0.000 claims 2
- 201000004502 glycogen storage disease II Diseases 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- LXBIFEVIBLOUGU-UHFFFAOYSA-N Deoxymannojirimycin Natural products OCC1NCC(O)C(O)C1O LXBIFEVIBLOUGU-UHFFFAOYSA-N 0.000 claims 1
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 238000011374 additional therapy Methods 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
- 108010049936 agalsidase alfa Proteins 0.000 claims 1
- 108010056760 agalsidase beta Proteins 0.000 claims 1
- 229940022705 aldurazyme Drugs 0.000 claims 1
- 229940049197 cerezyme Drugs 0.000 claims 1
- 229940014516 fabrazyme Drugs 0.000 claims 1
- 102000045921 human GAA Human genes 0.000 claims 1
- 108010039650 imiglucerase Proteins 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
- 229940103023 myozyme Drugs 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 229940099072 zavesca Drugs 0.000 claims 1
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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2150254A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2010525084A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2008245578A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2685332A1 (cg-RX-API-DMAC7.html) |
| IL (1) | IL201733A0 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2009011473A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2008134628A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| PL2533050T6 (pl) | 2006-05-16 | 2016-05-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry'ego |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| EP2252313B1 (en) | 2008-02-12 | 2015-04-08 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| WO2009155936A1 (en) | 2008-06-26 | 2009-12-30 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| WO2010056746A1 (en) * | 2008-11-11 | 2010-05-20 | 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 |
| ES2564093T3 (es) | 2009-04-09 | 2016-03-17 | Amicus Therapeutics, Inc. | Métodos para prevenir y/o tratar trastornos del almacenamiento lisosomal |
| 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 |
| LT2995306T (lt) | 2009-10-19 | 2019-04-10 | Amicus Therapeutics, Inc. | Naujos kompozicijos lizosomų kaupimosi sutrikimų profilaktikai ir (arba) gydymui |
| 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 |
| CA2778669C (en) * | 2009-10-27 | 2019-04-16 | Erytech Pharma | Composition to induce specific immune tolerance |
| ES2768995T3 (es) * | 2009-11-17 | 2020-06-24 | Baylor Res Institute | 1-desoxigalactonojirimicina para el uso en el tratamiento de una enfermedad cardíaca |
| NZ625712A (en) * | 2009-11-27 | 2016-02-26 | 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 |
| PT2646044T (pt) | 2010-11-30 | 2019-11-12 | Orphazyme As | Métodos para aumentar a atividade intracelular de hsp70 |
| SG10201604757RA (en) * | 2011-03-11 | 2016-08-30 | Amicus Therapeutics Inc | Dosing regimens for the treatment of fabry disease |
| EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
| EP2823043A4 (en) | 2012-03-07 | 2016-09-07 | Amicus Therapeutics Inc | HIGHLY CONCENTRATED ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF MORBUS POMPE |
| ES2924829T3 (es) | 2012-03-27 | 2022-10-11 | Amicus Therapeutics Inc | Compuestos novedosos para prevenir y/o tratar tesaurismosis lisosómicas y/o trastornos degenerativos del sistema nervioso central |
| HUE053565T2 (hu) * | 2012-05-03 | 2021-07-28 | Amicus Therapeutics Inc | Adagolási rendek Pompe betegség kezelésére |
| 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 |
| EP2874648A4 (en) * | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN |
| 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 |
| BR112017005810A2 (pt) | 2014-09-30 | 2018-02-27 | Amicus Therapeutics Inc | alfa-glucosidase ácida altamente potente com carboidratos melhorados |
| 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 |
| IL299470A (en) | 2015-12-30 | 2023-02-01 | Amicus Therapeutics Inc | Improved 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 |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| 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 |
| CA3023092A1 (en) | 2016-05-03 | 2017-11-09 | Sqz Biotechnologies Company | Intracellular delivery of biomolecules to induce tolerance |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| WO2018200618A1 (en) | 2017-04-25 | 2018-11-01 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders |
| LT3624831T (lt) | 2017-05-15 | 2023-07-10 | Amicus Therapeutics, Inc. | Rekombinantinė žmogaus rūgštinė alfa-glukozidazė |
| KR20240017110A (ko) * | 2017-05-30 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| HUE065615T2 (hu) * | 2017-05-30 | 2024-06-28 | Amicus Therapeutics Inc | Migalasztát, vesekárosodással szövõdött Fabry-kórban szenvedõ betegeg kezelésére |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| CN120044247A (zh) * | 2018-12-10 | 2025-05-27 | 戴纳立制药公司 | 溶酶体贮积症的生物标志物及其使用方法 |
| AU2020291002A1 (en) * | 2019-06-11 | 2022-01-06 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having renal impairment |
| MX2022001623A (es) | 2019-08-07 | 2022-03-02 | Amicus Therapeutics Inc | Metodos para tratar la enfermedad de fabry en pacientes que tienen una mutacion en el gen gla. |
| AU2021380947C1 (en) | 2020-11-19 | 2025-02-20 | Zevra Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
| 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 |
| 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 |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| JP2008545657A (ja) * | 2005-05-17 | 2008-12-18 | アミカス セラピューティックス インコーポレイテッド | １−デオキシノジリマイシンおよび誘導体を用いるポンペ病の治療方法 |
| 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 |
| US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
| EP2374451A3 (en) * | 2005-07-27 | 2012-01-25 | University of Florida Research Foundation, Inc. | Histone deacetylase inhibitors (HDAC) that correct protein misfolding and uses thereof |
| PL2533050T6 (pl) * | 2006-05-16 | 2016-05-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry'ego |
| 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 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
- 2008-04-28 AU AU2008245578A patent/AU2008245578A1/en not_active Abandoned
- 2008-04-28 MX MX2009011473A patent/MX2009011473A/es not_active Application Discontinuation
- 2008-04-28 US US12/597,238 patent/US9056101B2/en active Active
- 2008-04-28 EP EP08747020A patent/EP2150254A4/en not_active Withdrawn
- 2008-04-28 WO PCT/US2008/061764 patent/WO2008134628A2/en not_active Ceased
- 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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