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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP2150254A4 (OSRAM) |
| JP (1) | JP2010525084A (OSRAM) |
| AU (1) | AU2008245578A1 (OSRAM) |
| CA (1) | CA2685332A1 (OSRAM) |
| IL (1) | IL201733A0 (OSRAM) |
| MX (1) | MX2009011473A (OSRAM) |
| WO (1) | WO2008134628A2 (OSRAM) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2444102E (pt) | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
| FI3457135T3 (fi) | 2006-05-16 | 2025-03-08 | Amicus Therapeutics Inc | Fabryn taudin hoitovaihtoehtoja |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| HUE026543T2 (hu) | 2008-02-12 | 2016-06-28 | Amicus Therapeutics Inc | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| PL2659904T3 (pl) | 2008-06-26 | 2016-01-29 | Orphazyme Aps | Zastosowanie Hsp70 jako regulatora aktywności enzymatycznej |
| WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | 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 |
| CA2758271C (en) * | 2009-04-09 | 2018-06-05 | Robert Boyd | Methods for preventing and/or treating lysosomal storage disorders |
| WO2011049787A1 (en) * | 2009-10-19 | 2011-04-28 | Amicus Therapeutics, Inc. | Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases |
| RU2015147509A (ru) | 2009-10-19 | 2019-01-14 | Амикус Терапьютикс, Инк. | Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы |
| EP2490533B1 (en) | 2009-10-19 | 2015-09-16 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
| PT2493487T (pt) * | 2009-10-27 | 2016-11-08 | Erytech Pharma | Composição para induzir uma tolerância imunitária específica |
| US9066939B2 (en) * | 2009-11-17 | 2015-06-30 | Baylor Research Institute | Urinary triaosylceramide (GB3) as a marker of cardiac disease |
| ES2754398T3 (es) | 2009-11-27 | 2020-04-17 | Genzyme Corp | Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente |
| US8962564B2 (en) | 2010-11-08 | 2015-02-24 | Amicus Therapeutics, Inc. | Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity |
| DK2646044T3 (da) | 2010-11-30 | 2019-11-25 | Orphazyme As | Metoder til at øge den intracellulære aktivitet af 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 |
| KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| HK1205503A1 (en) | 2012-03-27 | 2015-12-18 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
| 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 |
| CA2917995C (en) | 2012-07-17 | 2021-01-26 | Amicus Therapeutics, Inc. | Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation |
| 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 |
| SI4273241T1 (sl) | 2014-09-30 | 2025-03-31 | Amicus Therapeutics, Inc. | Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati |
| 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 |
| US11613759B2 (en) | 2015-09-04 | 2023-03-28 | Sqz Biotechnologies Company | Intracellular delivery of biomolecules to cells comprising a cell wall |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| 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 |
| CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| NZ786723A (en) | 2016-03-30 | 2025-07-25 | Amicus Therapeutics Inc | Method for selection of high m6p recombinant proteins |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| LT3448382T (lt) | 2016-04-29 | 2021-04-12 | Orphazyme A/S | Arimoklomolis, skirtas su gliukocerebrozidaze susijusiems sutrikimams gydyti |
| AU2017259987B2 (en) | 2016-05-03 | 2023-10-19 | 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. |
| CN110573492A (zh) | 2017-04-25 | 2019-12-13 | 阿米库斯治疗学公司 | 用于预防和/或治疗中枢神经系统退行性失调和/或溶酶体贮积失调的新颖组合物 |
| DK3624831T5 (da) | 2017-05-15 | 2024-09-02 | Amicus Therapeutics Inc | Rekombinant human sur alfa-glucosidase |
| TWI795408B (zh) | 2017-05-30 | 2023-03-11 | 美商阿米庫斯醫療股份有限公司 | 治療有腎損傷的法布里患者的方法 |
| BR112019025083B1 (pt) | 2017-05-30 | 2022-08-09 | Amicus Therapeutics, Inc | Métodos de tratamento de pacientes portadores de fabry com insuficiência renal |
| CA3090496C (en) | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| CA3121927A1 (en) * | 2018-12-10 | 2020-06-18 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| AR120055A1 (es) * | 2019-06-11 | 2022-02-02 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal |
| CA3147055A1 (en) | 2019-08-07 | 2021-02-11 | Elfrida Benjamin | Methods of treating fabry disease in patients having a mutation in the gla gene |
| US20230416224A1 (en) | 2020-11-19 | 2023-12-28 | 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 |
| 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 |
| EP2932982B1 (en) * | 2005-05-17 | 2018-10-03 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| AU2006254796B2 (en) * | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
| AU2006272497B2 (en) * | 2005-07-27 | 2012-07-19 | University Of Florida Research Foundation, Inc. | Small compounds that correct protein misfolding and uses thereof |
| FI3457135T3 (fi) * | 2006-05-16 | 2025-03-08 | Amicus Therapeutics Inc | Fabryn taudin hoitovaihtoehtoja |
| WO2008121826A2 (en) * | 2007-03-30 | 2008-10-09 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease using pharmacological chaperones |
-
2008
- 2008-04-28 WO PCT/US2008/061764 patent/WO2008134628A2/en not_active Ceased
- 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 JP JP2010506557A patent/JP2010525084A/ja active Pending
- 2008-04-28 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
- 2008-04-28 MX MX2009011473A patent/MX2009011473A/es not_active Application Discontinuation
- 2008-04-28 AU AU2008245578A patent/AU2008245578A1/en not_active Abandoned
-
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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