JP2016163571A5 - - Google Patents
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- 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 (enExample) | 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 (enExample) |
| EP (4) | EP2946785B1 (enExample) |
| JP (7) | JP5844045B2 (enExample) |
| AU (7) | AU2009214648B2 (enExample) |
| CA (1) | CA2715407C (enExample) |
| CY (3) | CY1116466T1 (enExample) |
| DK (3) | DK2946785T3 (enExample) |
| ES (3) | ES2541933T3 (enExample) |
| HR (3) | HRP20150728T1 (enExample) |
| HU (3) | HUE042882T2 (enExample) |
| LT (2) | LT2946785T (enExample) |
| MX (1) | MX2010008835A (enExample) |
| PL (3) | PL3470077T3 (enExample) |
| PT (3) | PT3470077T (enExample) |
| SI (3) | SI3470077T1 (enExample) |
| WO (1) | WO2009102895A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3457135T3 (da) | 2006-05-16 | 2025-02-24 | Amicus Therapeutics Inc | Behandlingsmuligheder til fabrys sygdom |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| CA2718059A1 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for pompe disease |
| AR078498A1 (es) * | 2009-10-01 | 2011-11-09 | Baylor Res Inst | Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry |
| US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
| HRP20250058T1 (hr) | 2014-09-30 | 2025-03-28 | Amicus Therapeutics, Inc. | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
| 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 |
| EA201891507A1 (ru) | 2015-12-30 | 2018-12-28 | Амикус Терапьютикс, Инк. | Кислая альфа-глюкозидаза усиленного действия для лечения болезни помпе |
| LT3957320T (lt) | 2015-12-30 | 2023-12-11 | Amicus Therapeutics, Inc. | Patobulinta rūgštinė alfa-gliukozidazė pompe ligos gydymui |
| US10076514B2 (en) | 2016-03-22 | 2018-09-18 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
| TWI813388B (zh) | 2016-03-30 | 2023-08-21 | 美商阿米庫斯醫療股份有限公司 | 包含重組酸性α-葡萄糖苷酶之調配物 |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| 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 |
| CA3039673A1 (en) * | 2016-10-20 | 2018-04-26 | 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 |
| WO2018213340A1 (en) | 2017-05-15 | 2018-11-22 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| CA3224537C (en) | 2017-05-30 | 2025-09-09 | Amicus Therapeutics, Inc. | USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE |
| EP4327869B1 (en) | 2017-05-30 | 2025-12-10 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| KR102790184B1 (ko) | 2017-08-28 | 2025-04-01 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
| AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| 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 |
| US20210315875A1 (en) | 2018-08-20 | 2021-10-14 | 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 |
| TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
| CN114423428A (zh) * | 2019-08-07 | 2022-04-29 | 阿米库斯治疗学公司 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
| US20230220320A1 (en) | 2019-09-06 | 2023-07-13 | Amicus Therapeutics, Inc. | Method For Capturing And Purification Of Biologics |
| EP4114390B1 (en) * | 2020-03-06 | 2024-12-04 | 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 |
| WO2023288210A1 (en) | 2021-07-12 | 2023-01-19 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pediatric patients |
| WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| AU2023406510A1 (en) | 2022-12-02 | 2025-07-10 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| CN121057588A (zh) | 2022-12-02 | 2025-12-02 | 阿米库斯治疗学公司 | 用于在儿科患者中治疗迟发性庞贝病的方法 |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
| WO2026006625A1 (en) | 2024-06-26 | 2026-01-02 | Amicus Therapeutics, Inc. | Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease |
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| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| JP2020503900A (ja) | 2017-01-10 | 2020-02-06 | アミカス セラピューティックス インコーポレイテッド | ファブリー病の処置のための組換えアルファ−ガラクトシダーゼａ |
| CA3224537C (en) | 2017-05-30 | 2025-09-09 | Amicus Therapeutics, Inc. | USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| KR102790184B1 (ko) | 2017-08-28 | 2025-04-01 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| US20210315875A1 (en) | 2018-08-20 | 2021-10-14 | Amicus Therapeutics, Inc. | Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene |
| TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
| CN114423428A (zh) | 2019-08-07 | 2022-04-29 | 阿米库斯治疗学公司 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
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