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 (enExample) | 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 (enExample) |
| EP (1) | EP2150254A4 (enExample) |
| JP (1) | JP2010525084A (enExample) |
| AU (1) | AU2008245578A1 (enExample) |
| CA (1) | CA2685332A1 (enExample) |
| IL (1) | IL201733A0 (enExample) |
| MX (1) | MX2009011473A (enExample) |
| WO (1) | WO2008134628A2 (enExample) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004069190A2 (en) | 2003-01-31 | 2004-08-19 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| WO2007137072A2 (en) | 2006-05-16 | 2007-11-29 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| AU2009214648B2 (en) | 2008-02-12 | 2014-11-13 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| CA2728363C (en) | 2008-06-26 | 2019-02-19 | 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 |
| 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 |
| NZ595629A (en) | 2009-04-09 | 2013-04-26 | Amicus Therapeutics Inc | Methods for preventing and/or treating lysosomal storage disorders |
| MX337933B (es) | 2009-10-19 | 2016-03-29 | Amicus Therapeutics Inc | Nuevas composiciones para prevenir y/o tratar trastornos de almacenamiento lisosomal. |
| KR101769396B1 (ko) | 2009-10-19 | 2017-08-18 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물 |
| PL2490712T3 (pl) | 2009-10-19 | 2015-12-31 | Amicus Therapeutics Inc | Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz |
| PL2493487T3 (pl) * | 2009-10-27 | 2017-02-28 | Erytech Pharma | Kompozycja do indukowania swoistej tolerancji immunologicznej |
| WO2011063048A2 (en) * | 2009-11-17 | 2011-05-26 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
| 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 |
| DK2638152T3 (en) | 2010-11-08 | 2016-12-12 | Amicus Therapeutics Inc | VARIANTE, RECOMBINANT BETA-glucocerebrosidase PROTEINS WITH INCREASED STABILITY AND INCREASED RETAINED CATALYTIC ACTIVITY |
| PL2646044T3 (pl) | 2010-11-30 | 2020-03-31 | Orphazyme A/S | Sposoby zwiększenia aktywności wewnątrzkomórkowej Hsp70 |
| CN107088225A (zh) | 2011-03-11 | 2017-08-25 | 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 |
| KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| MX354127B (es) | 2012-03-27 | 2018-02-14 | Amicus Therapeutics Inc | Compuestos novedosos para prevenir y/o tratar tesaurismosis lisosomicas y/o trastornos degenerativos del sistema nervioso central. |
| ES2859761T3 (es) * | 2012-05-03 | 2021-10-04 | Amicus Therapeutics Inc | Pautas posológicas para el tratamiento de enfermedad de Pompe |
| 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 |
| WO2014014938A1 (en) | 2012-07-17 | 2014-01-23 | 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 |
| JP6678676B2 (ja) | 2014-09-15 | 2020-04-08 | オーファザイム エー／エス | アリモクロモル製剤 |
| DK3201320T5 (da) | 2014-09-30 | 2024-10-14 | Amicus Therapeutics Inc | Højpotent sur alfa-glucosidase med forbedrede carbohydrater |
| 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 |
| MX394968B (es) | 2015-12-30 | 2025-03-24 | Amicus Therapeutics Inc | Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. |
| 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 |
| KR102343162B1 (ko) | 2016-03-30 | 2021-12-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
| CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| BR112018070653A2 (pt) | 2016-04-29 | 2019-02-05 | Orphazyme As | ingrediente farmacêutico ativo, e, composição |
| KR102430856B1 (ko) | 2016-05-03 | 2022-08-08 | 에스큐지 바이오테크놀로지스 컴퍼니 | 관용을 유도하는 생체분자의 세포내 전달 |
| 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 |
| SMT202300187T1 (it) | 2017-05-15 | 2023-07-20 | Amicus Therapeutics Inc | Alfa-glucosidasi acida umana ricombinante |
| 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 |
| CA3224529A1 (en) * | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| CA3090496C (en) | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| US20220025065A1 (en) * | 2018-12-10 | 2022-01-27 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| US20220313670A1 (en) * | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| US11833164B2 (en) | 2019-08-07 | 2023-12-05 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| 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 |
| WO2004069190A2 (en) * | 2003-01-31 | 2004-08-19 | 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 |
| ES2572148T3 (es) * | 2005-05-17 | 2016-05-30 | Amicus Therapeutics Inc | Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados |
| 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 |
| EP1909812A4 (en) * | 2005-07-27 | 2009-11-25 | Univ Florida | SMALL CONNECTIONS FOR THE CORRECTION OF PROTEIN MISFAIRINGS AND USES THEREOF |
| WO2007137072A2 (en) * | 2006-05-16 | 2007-11-29 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| US20100113517A1 (en) * | 2007-03-30 | 2010-05-06 | 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 EP EP08747020A patent/EP2150254A4/en not_active Withdrawn
- 2008-04-28 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
- 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 JP JP2010506557A patent/JP2010525084A/ja active Pending
- 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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