JP2014528901A5 - - Google Patents
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- Publication number
- JP2014528901A5 JP2014528901A5 JP2013557862A JP2013557862A JP2014528901A5 JP 2014528901 A5 JP2014528901 A5 JP 2014528901A5 JP 2013557862 A JP2013557862 A JP 2013557862A JP 2013557862 A JP2013557862 A JP 2013557862A JP 2014528901 A5 JP2014528901 A5 JP 2014528901A5
- Authority
- JP
- Japan
- Prior art keywords
- deoxygalactonojirimycin
- gal
- administration
- replacement therapy
- enzyme replacement
- 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
- 239000003814 drug Substances 0.000 claims 14
- 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 14
- 229950007469 migalastat Drugs 0.000 claims 11
- 238000002641 enzyme replacement therapy Methods 0.000 claims 10
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 claims 8
- NNISLDGFPWIBDF-MPRBLYSKSA-N alpha-D-Gal-(1->3)-beta-D-Gal-(1->4)-D-GlcNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)C(O)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@@H](CO)O1 NNISLDGFPWIBDF-MPRBLYSKSA-N 0.000 claims 8
- 208000024720 Fabry Disease Diseases 0.000 claims 2
- QCQCHGYLTSGIGX-GHXANHINSA-N 4-[[(3ar,5ar,5br,7ar,9s,11ar,11br,13as)-5a,5b,8,8,11a-pentamethyl-3a-[(5-methylpyridine-3-carbonyl)amino]-2-oxo-1-propan-2-yl-4,5,6,7,7a,9,10,11,11b,12,13,13a-dodecahydro-3h-cyclopenta[a]chrysen-9-yl]oxy]-2,2-dimethyl-4-oxobutanoic acid Chemical compound N([C@@]12CC[C@@]3(C)[C@]4(C)CC[C@H]5C(C)(C)[C@@H](OC(=O)CC(C)(C)C(O)=O)CC[C@]5(C)[C@H]4CC[C@@H]3C1=C(C(C2)=O)C(C)C)C(=O)C1=CN=CC(C)=C1 QCQCHGYLTSGIGX-GHXANHINSA-N 0.000 claims 1
- YQYJSBFKSSDGFO-UHFFFAOYSA-N Epihygromycin Natural products OC1C(O)C(C(=O)C)OC1OC(C(=C1)O)=CC=C1C=C(C)C(=O)NC1C(O)C(O)C2OCOC2C1O YQYJSBFKSSDGFO-UHFFFAOYSA-N 0.000 claims 1
- 238000002648 combination therapy Methods 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161451798P | 2011-03-11 | 2011-03-11 | |
| US61/451,798 | 2011-03-11 | ||
| US201161578201P | 2011-12-20 | 2011-12-20 | |
| US61/578,201 | 2011-12-20 | ||
| US201261596165P | 2012-02-07 | 2012-02-07 | |
| US61/596,165 | 2012-02-07 | ||
| PCT/US2012/028260 WO2012125402A2 (en) | 2011-03-11 | 2012-03-08 | Dosing regimens for the treatment of fabry disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017038469A Division JP2017132780A (ja) | 2011-03-11 | 2017-03-01 | ファブリー病の治療用投薬レジメン |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2014528901A JP2014528901A (ja) | 2014-10-30 |
| JP2014528901A5 true JP2014528901A5 (enExample) | 2015-04-30 |
Family
ID=46831259
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013557862A Pending JP2014528901A (ja) | 2011-03-11 | 2012-03-08 | ファブリー病の治療用投薬レジメン |
| JP2017038469A Pending JP2017132780A (ja) | 2011-03-11 | 2017-03-01 | ファブリー病の治療用投薬レジメン |
| JP2019186952A Pending JP2020033360A (ja) | 2011-03-11 | 2019-10-10 | ファブリー病の治療用投薬レジメン |
| JP2021018456A Withdrawn JP2021098697A (ja) | 2011-03-11 | 2021-02-08 | ファブリー病の治療用投薬レジメン |
| JP2023045001A Pending JP2023093448A (ja) | 2011-03-11 | 2023-03-22 | ファブリー病の治療用投薬レジメン |
| JP2025179237A Pending JP2026035578A (ja) | 2011-03-11 | 2025-10-24 | ファブリー病の治療用投薬レジメン |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017038469A Pending JP2017132780A (ja) | 2011-03-11 | 2017-03-01 | ファブリー病の治療用投薬レジメン |
| JP2019186952A Pending JP2020033360A (ja) | 2011-03-11 | 2019-10-10 | ファブリー病の治療用投薬レジメン |
| JP2021018456A Withdrawn JP2021098697A (ja) | 2011-03-11 | 2021-02-08 | ファブリー病の治療用投薬レジメン |
| JP2023045001A Pending JP2023093448A (ja) | 2011-03-11 | 2023-03-22 | ファブリー病の治療用投薬レジメン |
| JP2025179237A Pending JP2026035578A (ja) | 2011-03-11 | 2025-10-24 | ファブリー病の治療用投薬レジメン |
Country Status (16)
| Country | Link |
|---|---|
| US (4) | US20140219986A1 (enExample) |
| EP (2) | EP2683382B1 (enExample) |
| JP (6) | JP2014528901A (enExample) |
| KR (1) | KR20140011367A (enExample) |
| CN (2) | CN107088225A (enExample) |
| AU (6) | AU2012229330B2 (enExample) |
| CA (1) | CA2829947C (enExample) |
| CL (1) | CL2013002601A1 (enExample) |
| EA (1) | EA031874B1 (enExample) |
| ES (1) | ES2807502T3 (enExample) |
| HK (1) | HK1242998A1 (enExample) |
| MX (1) | MX2013010446A (enExample) |
| SG (2) | SG10201604757RA (enExample) |
| TW (3) | TW201740945A (enExample) |
| WO (1) | WO2012125402A2 (enExample) |
| ZA (1) | ZA201306735B (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| UY34317A (es) | 2011-09-12 | 2013-02-28 | Genzyme Corp | Anticuerpo antireceptor de célula T (alfa)/ß |
| US9790268B2 (en) | 2012-09-12 | 2017-10-17 | Genzyme Corporation | Fc containing polypeptides with altered glycosylation and reduced effector function |
| US9580511B2 (en) | 2013-03-11 | 2017-02-28 | Genzyme Corporation | Site-specific antibody-drug conjugation through glycoengineering |
| CN106687596A (zh) * | 2014-02-17 | 2017-05-17 | 葛兰素史密斯克莱知识产权发展有限公司 | 微生物学方法 |
| HUE061339T2 (hu) | 2014-03-19 | 2023-06-28 | Genzyme Corp | Targetáló csoportok helyspecifikus glikotechnikai átalakítása |
| US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| ES3067417T3 (en) | 2015-08-11 | 2026-05-21 | Cognoa Inc | Apparatus to determine developmental progress with artificial intelligence and user input |
| US11972336B2 (en) | 2015-12-18 | 2024-04-30 | Cognoa, Inc. | Machine learning platform and system for data analysis |
| 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 |
| WO2018017721A1 (en) * | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| CN118609834A (zh) | 2016-11-14 | 2024-09-06 | 科格诺亚公司 | 用于评估发育状况并提供覆盖度和可靠性控制的方法和装置 |
| JP7324709B2 (ja) * | 2017-02-09 | 2023-08-10 | コグノア，インク． | デジタル個別化医療のためのプラットフォームとシステム |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| PL4062916T3 (pl) * | 2017-05-30 | 2025-03-17 | Amicus Therapeutics, Inc. | Leczenie pacjentów z chorobą Fabry’ego mających pewne mutacje |
| JP7066157B2 (ja) | 2017-06-14 | 2022-05-13 | 学校法人 明治薬科大学 | ファブリー病治療用医薬の組合せ物及びその利用 |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| CN113873935B (zh) | 2019-03-22 | 2024-11-22 | 科格诺亚公司 | 个性化数字化治疗方法和装置 |
| JP7575401B2 (ja) | 2019-04-03 | 2024-10-29 | ジェンザイム・コーポレーション | 断片化が低減した抗アルファベータｔｃｒ結合ポリペプチド |
| US20220313670A1 (en) * | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| WO2021046412A1 (en) | 2019-09-06 | 2021-03-11 | Cognoa, Inc. | Methods, systems, and devices for the diagnosis of behavioral disorders, developmental delays, and neurologic impairments |
| FI4114390T3 (fi) * | 2020-03-06 | 2025-03-07 | Amicus Therapeutics Inc | Menetelmiä fabryn taudin hoitamiseksi potilaissa, joilla on mutaatio gla-geenissä |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| US20240120050A1 (en) * | 2022-10-07 | 2024-04-11 | Insight Direct Usa, Inc. | Machine learning method for predicting a health outcome of a patient using video and audio analytics |
| CN118488839A (zh) * | 2022-12-13 | 2024-08-13 | 阿米库斯治疗学公司 | 改善米加司他的药代动力学的方法 |
| US20240197706A1 (en) * | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
| TR2024011149A1 (tr) * | 2024-08-23 | 2026-03-23 | Sanovel Ilac Sanayi Ve Ticaret Anonim Sirketi | Mi̇galastat i̇çeren kapsül formülasyonlari |
| TR2024011147A1 (tr) * | 2024-08-23 | 2026-03-23 | Sanovel Ilac Sanayi Ve Ticaret Anonim Sirketi | Mi̇galastat i̇çeren kapsül formülasyonu |
| US20260188467A1 (en) * | 2024-12-27 | 2026-07-02 | Center for Social Dynamics, LLC | Parallel Processing Systems and Methods For Real-Time Multimodal Behavioral Classification Using Neural Networks With Dynamic Content Adaptation |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4551177A (en) * | 1984-04-23 | 1985-11-05 | National Starch And Chemical Corporation | Compressible starches as binders for tablets or capsules |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| JP5589165B2 (ja) * | 2003-01-31 | 2014-09-17 | マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー | タンパク質欠損性障害の治療のための併用療法 |
| US7879354B2 (en) * | 2004-01-13 | 2011-02-01 | Mcneil-Ppc, Inc. | Rapidly disintegrating gelatinous coated tablets |
| CN100457106C (zh) * | 2005-06-15 | 2009-02-04 | 同济大学 | 1-脱氧野尻霉素在制备治疗糖尿病肾病药物中的应用 |
| MX351004B (es) * | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| EP2142197A4 (en) * | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES |
| AU2008245578A1 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| US20100291060A1 (en) * | 2007-08-29 | 2010-11-18 | Shire Human Genetic Therapies, Inc | Subcutaneous administration of alpha-galactosidase a |
-
2012
- 2012-03-08 WO PCT/US2012/028260 patent/WO2012125402A2/en not_active Ceased
- 2012-03-08 KR KR1020137026394A patent/KR20140011367A/ko not_active Ceased
- 2012-03-08 SG SG10201604757RA patent/SG10201604757RA/en unknown
- 2012-03-08 CN CN201611235913.7A patent/CN107088225A/zh active Pending
- 2012-03-08 EP EP12758017.3A patent/EP2683382B1/en active Active
- 2012-03-08 CA CA2829947A patent/CA2829947C/en active Active
- 2012-03-08 AU AU2012229330A patent/AU2012229330B2/en active Active
- 2012-03-08 ES ES12758017T patent/ES2807502T3/es active Active
- 2012-03-08 EA EA201301018A patent/EA031874B1/ru not_active IP Right Cessation
- 2012-03-08 US US14/004,335 patent/US20140219986A1/en not_active Abandoned
- 2012-03-08 SG SG2013067954A patent/SG193379A1/en unknown
- 2012-03-08 MX MX2013010446A patent/MX2013010446A/es unknown
- 2012-03-08 CN CN201280021877.8A patent/CN103974619B/zh active Active
- 2012-03-08 JP JP2013557862A patent/JP2014528901A/ja active Pending
- 2012-03-08 EP EP20160164.8A patent/EP3698792A1/en active Pending
- 2012-03-12 TW TW106126844A patent/TW201740945A/zh unknown
- 2012-03-12 TW TW101108345A patent/TWI624258B/zh not_active IP Right Cessation
- 2012-03-12 TW TW105118425A patent/TWI624259B/zh not_active IP Right Cessation
-
2013
- 2013-09-09 ZA ZA2013/06735A patent/ZA201306735B/en unknown
- 2013-09-10 CL CL2013002601A patent/CL2013002601A1/es unknown
-
2016
- 2016-06-28 AU AU2016204445A patent/AU2016204445B2/en active Active
-
2017
- 2017-03-01 JP JP2017038469A patent/JP2017132780A/ja active Pending
- 2017-10-12 US US15/782,431 patent/US20180153999A1/en not_active Abandoned
-
2018
- 2018-02-23 HK HK18102615.1A patent/HK1242998A1/zh unknown
- 2018-03-07 AU AU2018201637A patent/AU2018201637A1/en not_active Abandoned
-
2019
- 2019-10-10 JP JP2019186952A patent/JP2020033360A/ja active Pending
-
2020
- 2020-03-02 US US16/806,404 patent/US20200268890A1/en not_active Abandoned
- 2020-03-09 AU AU2020201715A patent/AU2020201715A1/en not_active Abandoned
-
2021
- 2021-02-08 JP JP2021018456A patent/JP2021098697A/ja not_active Withdrawn
-
2022
- 2022-05-05 AU AU2022203011A patent/AU2022203011A1/en not_active Abandoned
-
2023
- 2023-03-22 JP JP2023045001A patent/JP2023093448A/ja active Pending
- 2023-07-17 US US18/222,745 patent/US20240115708A1/en active Pending
-
2024
- 2024-07-31 AU AU2024205251A patent/AU2024205251A1/en active Pending
-
2025
- 2025-10-24 JP JP2025179237A patent/JP2026035578A/ja active Pending
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