JP2016163571A5 - - Google Patents
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- Publication number
- JP2016163571A5 JP2016163571A5 JP2016039171A JP2016039171A JP2016163571A5 JP 2016163571 A5 JP2016163571 A5 JP 2016163571A5 JP 2016039171 A JP2016039171 A JP 2016039171A JP 2016039171 A JP2016039171 A JP 2016039171A JP 2016163571 A5 JP2016163571 A5 JP 2016163571A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- galactosidase
- specific pharmacological
- pharmacological chaperone
- gala
- 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
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- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 102100026277 Alpha-galactosidase A Human genes 0.000 claims 4
- 108010006519 Molecular Chaperones Proteins 0.000 claims 4
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 4
- 230000000144 pharmacologic effect Effects 0.000 claims 4
- 102200077160 rs797044774 Human genes 0.000 claims 3
- 102220486257 Serine hydroxymethyltransferase, cytosolic_N320I_mutation Human genes 0.000 claims 2
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 claims 2
- 229950007469 migalastat Drugs 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 102220152464 rs144140791 Human genes 0.000 claims 2
- 102220085241 rs879253808 Human genes 0.000 claims 2
- 102220480233 Adhesion G-protein coupled receptor D1_P293A_mutation Human genes 0.000 claims 1
- 102220486386 Alpha-galactosidase A_A288D_mutation Human genes 0.000 claims 1
- 102220486228 Alpha-galactosidase A_G35R_mutation Human genes 0.000 claims 1
- 102220486653 Alpha-galactosidase A_I242N_mutation Human genes 0.000 claims 1
- 102220486151 Alpha-galactosidase A_M72V_mutation Human genes 0.000 claims 1
- 102220486311 Alpha-galactosidase A_P265R_mutation Human genes 0.000 claims 1
- 102220486351 Alpha-galactosidase A_Q280H_mutation Human genes 0.000 claims 1
- 102220486115 Alpha-galactosidase A_R49L_mutation Human genes 0.000 claims 1
- 102220486129 Alpha-galactosidase A_R49S_mutation Human genes 0.000 claims 1
- 102220481462 Alpha-galactosidase A_V316E_mutation Human genes 0.000 claims 1
- 102220486015 Alpha-galactosidase A_W95S_mutation Human genes 0.000 claims 1
- 102220466993 Annexin A3_I219N_mutation Human genes 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 102220514548 Somatostatin receptor type 1_N34K_mutation Human genes 0.000 claims 1
- 102200076863 c.119C>T Human genes 0.000 claims 1
- 102200076464 c.272T>C Human genes 0.000 claims 1
- 102220362014 c.702C>G Human genes 0.000 claims 1
- 102200077268 c.826A>G Human genes 0.000 claims 1
- 102200077291 c.893A>G Human genes 0.000 claims 1
- 102200076847 c.95T>C Human genes 0.000 claims 1
- 102200077287 rs104894830 Human genes 0.000 claims 1
- 102200076873 rs104894831 Human genes 0.000 claims 1
- 102200077288 rs104894846 Human genes 0.000 claims 1
- 102200076840 rs104894847 Human genes 0.000 claims 1
- 102200065199 rs1057515579 Human genes 0.000 claims 1
- 102200076919 rs1057521047 Human genes 0.000 claims 1
- 102200010382 rs1057521062 Human genes 0.000 claims 1
- 102200077177 rs111422676 Human genes 0.000 claims 1
- 102200076472 rs113173389 Human genes 0.000 claims 1
- 102200069129 rs121434329 Human genes 0.000 claims 1
- 102200005231 rs121908280 Human genes 0.000 claims 1
- 102200023312 rs121912940 Human genes 0.000 claims 1
- 102200158224 rs121918530 Human genes 0.000 claims 1
- 102200063052 rs137852853 Human genes 0.000 claims 1
- 102200077273 rs140329381 Human genes 0.000 claims 1
- 102220104213 rs140506267 Human genes 0.000 claims 1
- 102220141945 rs140804035 Human genes 0.000 claims 1
- 102200036676 rs143947056 Human genes 0.000 claims 1
- 102220260525 rs1554097567 Human genes 0.000 claims 1
- 102200049940 rs1554402092 Human genes 0.000 claims 1
- 102200070822 rs1555710077 Human genes 0.000 claims 1
- 102200077127 rs1555984869 Human genes 0.000 claims 1
- 102200094055 rs17576 Human genes 0.000 claims 1
- 102200077342 rs190347120 Human genes 0.000 claims 1
- 102220228444 rs200183599 Human genes 0.000 claims 1
- 102220007476 rs267606804 Human genes 0.000 claims 1
- 102200077267 rs28935485 Human genes 0.000 claims 1
- 102220087827 rs28935489 Human genes 0.000 claims 1
- 102220082624 rs370063423 Human genes 0.000 claims 1
- 102200078669 rs370595480 Human genes 0.000 claims 1
- 102220214294 rs373488862 Human genes 0.000 claims 1
- 102200013203 rs387906468 Human genes 0.000 claims 1
- 102200129586 rs397514731 Human genes 0.000 claims 1
- 102200018556 rs587779420 Human genes 0.000 claims 1
- 102200020122 rs6054 Human genes 0.000 claims 1
- 102200076593 rs727503948 Human genes 0.000 claims 1
- 102200077186 rs727504348 Human genes 0.000 claims 1
- 102220038571 rs72937434 Human genes 0.000 claims 1
- 102200077248 rs730880451 Human genes 0.000 claims 1
- 102220277657 rs746045584 Human genes 0.000 claims 1
- 102200007201 rs750496798 Human genes 0.000 claims 1
- 102200068463 rs760106433 Human genes 0.000 claims 1
- 102200065197 rs760277934 Human genes 0.000 claims 1
- 102220064075 rs762381137 Human genes 0.000 claims 1
- 102220114730 rs763889166 Human genes 0.000 claims 1
- 102220166398 rs766522793 Human genes 0.000 claims 1
- 102220200219 rs770497192 Human genes 0.000 claims 1
- 102220272713 rs777653224 Human genes 0.000 claims 1
- 102200076503 rs782197638 Human genes 0.000 claims 1
- 102220075588 rs796053298 Human genes 0.000 claims 1
- 102220055601 rs797044497 Human genes 0.000 claims 1
- 102200160956 rs869320708 Human genes 0.000 claims 1
- 102200136953 rs879253837 Human genes 0.000 claims 1
- 102200037543 rs879254797 Human genes 0.000 claims 1
- 102220456815 rs886051894 Human genes 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US2814108P | 2008-02-12 | 2008-02-12 | |
| US61/028,141 | 2008-02-12 | ||
| US3568408P | 2008-03-11 | 2008-03-11 | |
| US61/035,684 | 2008-03-11 | ||
| US9363108P | 2008-09-02 | 2008-09-02 | |
| US61/093,631 | 2008-09-02 | ||
| US11349608P | 2008-11-11 | 2008-11-11 | |
| US61/113,496 | 2008-11-11 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014244283A Division JP2015091239A (ja) | 2008-02-12 | 2014-12-02 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018245609A Division JP6837469B2 (ja) | 2008-02-12 | 2018-12-27 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016163571A JP2016163571A (ja) | 2016-09-08 |
| JP2016163571A5 true JP2016163571A5 (cg-RX-API-DMAC7.html) | 2016-12-01 |
| JP6672013B2 JP6672013B2 (ja) | 2020-03-25 |
Family
ID=40957500
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010546901A Active JP5844045B2 (ja) | 2008-02-12 | 2009-02-12 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2014244283A Pending JP2015091239A (ja) | 2008-02-12 | 2014-12-02 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2016039171A Active JP6672013B2 (ja) | 2008-02-12 | 2016-03-01 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2018245609A Active JP6837469B2 (ja) | 2008-02-12 | 2018-12-27 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2021019383A Active JP7277493B2 (ja) | 2008-02-12 | 2021-02-09 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2023076515A Withdrawn JP2023109807A (ja) | 2008-02-12 | 2023-05-08 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2025097940A Pending JP2025148351A (ja) | 2008-02-12 | 2025-06-11 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010546901A Active JP5844045B2 (ja) | 2008-02-12 | 2009-02-12 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2014244283A Pending JP2015091239A (ja) | 2008-02-12 | 2014-12-02 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018245609A Active JP6837469B2 (ja) | 2008-02-12 | 2018-12-27 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2021019383A Active JP7277493B2 (ja) | 2008-02-12 | 2021-02-09 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2023076515A Withdrawn JP2023109807A (ja) | 2008-02-12 | 2023-05-08 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
| JP2025097940A Pending JP2025148351A (ja) | 2008-02-12 | 2025-06-11 | 疾病の薬理シャペロン治療に対する応答性を予測する方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (cg-RX-API-DMAC7.html) |
| EP (4) | EP3470077B1 (cg-RX-API-DMAC7.html) |
| JP (7) | JP5844045B2 (cg-RX-API-DMAC7.html) |
| AU (7) | AU2009214648B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2715407C (cg-RX-API-DMAC7.html) |
| CY (3) | CY1116466T1 (cg-RX-API-DMAC7.html) |
| DK (3) | DK3470077T3 (cg-RX-API-DMAC7.html) |
| ES (3) | ES2836121T3 (cg-RX-API-DMAC7.html) |
| HR (3) | HRP20150728T1 (cg-RX-API-DMAC7.html) |
| HU (3) | HUE042882T2 (cg-RX-API-DMAC7.html) |
| LT (2) | LT3470077T (cg-RX-API-DMAC7.html) |
| MX (1) | MX2010008835A (cg-RX-API-DMAC7.html) |
| PL (3) | PL2252313T3 (cg-RX-API-DMAC7.html) |
| PT (3) | PT2946785T (cg-RX-API-DMAC7.html) |
| SI (3) | SI2252313T1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2009102895A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2533050T6 (da) | 2006-05-16 | 2015-09-21 | Amicus Therapeutics Inc | Behandlingsmuligheder ved Fabrys sygdom |
| HUE042882T2 (hu) | 2008-02-12 | 2019-07-29 | Amicus Therapeutics Inc | Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| JP2011517556A (ja) * | 2008-03-12 | 2011-06-16 | アミカス セラピューティックス インコーポレイテッド | ポンペ病のための治療選択肢を診断し評価するための検定 |
| WO2011041718A2 (en) * | 2009-10-01 | 2011-04-07 | Baylor Research Institute | Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease |
| US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
| CA2961762C (en) | 2014-09-30 | 2024-03-05 | Amicus Therapeutics, Inc. | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| AU2016233135B2 (en) * | 2015-03-19 | 2021-07-08 | Translate Bio, Inc. | mRNA therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| DK3957320T5 (da) | 2015-12-30 | 2024-08-05 | Amicus Therapeutics Inc | Forstærket syre-alfa-glucosidase til behandling af pompes sygdom |
| MY198085A (en) | 2015-12-30 | 2023-07-31 | Amicus Therapeutics Inc | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
| US10227577B2 (en) * | 2016-03-30 | 2019-03-12 | Amicus Therapeutics, Inc. | Method for selection of high M6P recombinant proteins |
| LT3436053T (lt) | 2016-03-30 | 2022-07-11 | Amicus Therapeutics, Inc. | Kompozicijos, apimančios rekombinantinę rūgštinę alfa-gliukozidazę |
| KR102618519B1 (ko) | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| KR20240017126A (ko) | 2016-07-19 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료 |
| JP6546135B2 (ja) | 2016-08-24 | 2019-07-17 | 株式会社ノリタケカンパニーリミテド | 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット |
| EP3528852B1 (en) * | 2016-10-20 | 2025-09-03 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| HRP20230576T1 (hr) | 2017-05-15 | 2023-09-01 | Amicus Therapeutics, Inc. | Rekombinantna ljudska kisela alfa-glukozidaza |
| BR112019025083B1 (pt) | 2017-05-30 | 2022-08-09 | Amicus Therapeutics, Inc | Métodos de tratamento de pacientes portadores de fabry com insuficiência renal |
| HUE069837T2 (hu) | 2017-05-30 | 2025-04-28 | Amicus Therapeutics Inc | Bizonyos mutációkat hordozó Fabry-kóros betegek kezelése |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| IL310648A (en) | 2017-08-28 | 2024-04-01 | Amicus Therapeutics Inc | Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease |
| KR20200128675A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 전형적 파브리병 환자의 치료 |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| EP4299117A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
| TW202042812A (zh) | 2019-01-22 | 2020-12-01 | 美商阿米庫斯醫療股份有限公司 | 減少法布瑞氏症患者中之腦血管事件之方法 |
| EA202290024A1 (ru) | 2019-06-11 | 2022-03-14 | Амикус Терапьютикс, Инк. | Способы лечения болезни фабри у пациентов с почечной недостаточностью |
| CA3147055A1 (en) | 2019-08-07 | 2021-02-11 | Elfrida Benjamin | Methods of treating fabry disease in patients having a mutation in the gla gene |
| WO2021046443A1 (en) | 2019-09-06 | 2021-03-11 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
| US20230136297A1 (en) * | 2020-03-06 | 2023-05-04 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having A Mutation In The GLA Gene |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| KR20240034203A (ko) | 2021-07-12 | 2024-03-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 소아청소년 환자에서 파브리병을 치료하는 방법 |
| JP2025515159A (ja) | 2022-05-05 | 2025-05-13 | アミカス セラピューティックス インコーポレイテッド | ポンペ病の治療方法 |
| AU2023406479A1 (en) | 2022-12-02 | 2025-07-03 | Amicus Therapeutics, Inc. | Methods for treating late onset pompe disease in pediatric patients |
| TW202436620A (zh) | 2022-12-02 | 2024-09-16 | 美商阿米庫斯醫療股份有限公司 | 用於在兒科患者中治療嬰兒型龐貝氏症之方法 |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
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| EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
| KR20240017126A (ko) | 2016-07-19 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료 |
| EP3568152A1 (en) | 2017-01-10 | 2019-11-20 | Amicus Therapeutics, Inc. | Recombinant alpha-galactosidase a for treatment of fabry disease |
| BR112019025083B1 (pt) | 2017-05-30 | 2022-08-09 | Amicus Therapeutics, Inc | Métodos de tratamento de pacientes portadores de fabry com insuficiência renal |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| IL310648A (en) | 2017-08-28 | 2024-04-01 | Amicus Therapeutics Inc | Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| KR20200128675A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 전형적 파브리병 환자의 치료 |
| EP4299117A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| EA202290024A1 (ru) | 2019-06-11 | 2022-03-14 | Амикус Терапьютикс, Инк. | Способы лечения болезни фабри у пациентов с почечной недостаточностью |
| CA3147055A1 (en) | 2019-08-07 | 2021-02-11 | Elfrida Benjamin | Methods of treating fabry disease in patients having a mutation in the gla gene |
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