JP2015519329A5 - - Google Patents
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- Publication number
- JP2015519329A5 JP2015519329A5 JP2015510446A JP2015510446A JP2015519329A5 JP 2015519329 A5 JP2015519329 A5 JP 2015519329A5 JP 2015510446 A JP2015510446 A JP 2015510446A JP 2015510446 A JP2015510446 A JP 2015510446A JP 2015519329 A5 JP2015519329 A5 JP 2015519329A5
- Authority
- JP
- Japan
- Prior art keywords
- deoxynojirimycin
- glucosidase enzyme
- acid
- enzyme replacement
- replacement therapy
- 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
Links
- LXBIFEVIBLOUGU-JGWLITMVSA-N duvoglustat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O LXBIFEVIBLOUGU-JGWLITMVSA-N 0.000 claims 52
- 239000003814 drug Substances 0.000 claims 35
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 27
- 239000002253 acid Substances 0.000 claims 27
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 27
- 238000002641 enzyme replacement therapy Methods 0.000 claims 22
- LXBIFEVIBLOUGU-UHFFFAOYSA-N Deoxymannojirimycin Natural products OCC1NCC(O)C(O)C1O LXBIFEVIBLOUGU-UHFFFAOYSA-N 0.000 claims 18
- 239000003795 chemical substances by application Substances 0.000 claims 8
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 5
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 5
- 201000004502 glycogen storage disease II Diseases 0.000 claims 5
- 229940124597 therapeutic agent Drugs 0.000 claims 5
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 2
- 238000002648 combination therapy Methods 0.000 claims 2
- 102000045921 human GAA Human genes 0.000 claims 2
- ZJIHMALTJRDNQI-VFQQELCFSA-N (2r,3r,4r,5s)-2-(hydroxymethyl)piperidine-3,4,5-triol;hydrochloride Chemical compound Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O ZJIHMALTJRDNQI-VFQQELCFSA-N 0.000 claims 1
- AGOYDEPGAOXOCK-KCBOHYOISA-N clarithromycin Chemical class O([C@@H]1[C@@H](C)C(=O)O[C@@H]([C@@]([C@H](O)[C@@H](C)C(=O)[C@H](C)C[C@](C)([C@H](O[C@H]2[C@@H]([C@H](C[C@@H](C)O2)N(C)C)O)[C@H]1C)OC)(C)O)CC)[C@H]1C[C@@](C)(OC)[C@@H](O)[C@H](C)O1 AGOYDEPGAOXOCK-KCBOHYOISA-N 0.000 claims 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261642311P | 2012-05-03 | 2012-05-03 | |
| US61/642,311 | 2012-05-03 | ||
| US201261664011P | 2012-06-25 | 2012-06-25 | |
| US61/664,011 | 2012-06-25 | ||
| US201261697179P | 2012-09-05 | 2012-09-05 | |
| US61/697,179 | 2012-09-05 | ||
| US201361749132P | 2013-01-04 | 2013-01-04 | |
| US201361749234P | 2013-01-04 | 2013-01-04 | |
| US61/749,234 | 2013-01-04 | ||
| US61/749,132 | 2013-01-04 | ||
| PCT/US2013/039215 WO2013166249A1 (en) | 2012-05-03 | 2013-05-02 | Dosing regimens for the treatment of pompe disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018007672A Division JP6612370B2 (ja) | 2012-05-03 | 2018-01-19 | ポンペ病の処置のための投与計画 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015519329A JP2015519329A (ja) | 2015-07-09 |
| JP2015519329A5 true JP2015519329A5 (enExample) | 2018-03-08 |
| JP6381521B2 JP6381521B2 (ja) | 2018-08-29 |
Family
ID=49514881
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015510446A Active JP6381521B2 (ja) | 2012-05-03 | 2013-05-02 | ポンペ病の処置のための投与計画 |
| JP2018007672A Active JP6612370B2 (ja) | 2012-05-03 | 2018-01-19 | ポンペ病の処置のための投与計画 |
| JP2019196583A Active JP6876111B2 (ja) | 2012-05-03 | 2019-10-29 | ポンペ病の処置のための投与計画 |
| JP2021010931A Active JP7046241B2 (ja) | 2012-05-03 | 2021-01-27 | ポンペ病の処置のための投与計画 |
| JP2022044812A Withdrawn JP2022101544A (ja) | 2012-05-03 | 2022-03-22 | ポンペ病の処置のための投与計画 |
| JP2024034587A Pending JP2024081667A (ja) | 2012-05-03 | 2024-03-07 | ポンペ病の処置のための投与計画 |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018007672A Active JP6612370B2 (ja) | 2012-05-03 | 2018-01-19 | ポンペ病の処置のための投与計画 |
| JP2019196583A Active JP6876111B2 (ja) | 2012-05-03 | 2019-10-29 | ポンペ病の処置のための投与計画 |
| JP2021010931A Active JP7046241B2 (ja) | 2012-05-03 | 2021-01-27 | ポンペ病の処置のための投与計画 |
| JP2022044812A Withdrawn JP2022101544A (ja) | 2012-05-03 | 2022-03-22 | ポンペ病の処置のための投与計画 |
| JP2024034587A Pending JP2024081667A (ja) | 2012-05-03 | 2024-03-07 | ポンペ病の処置のための投与計画 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20150086530A1 (enExample) |
| EP (3) | EP2844279B1 (enExample) |
| JP (6) | JP6381521B2 (enExample) |
| CY (1) | CY1124135T1 (enExample) |
| DK (1) | DK2844279T3 (enExample) |
| ES (2) | ES2980828T3 (enExample) |
| HR (1) | HRP20210398T1 (enExample) |
| HU (1) | HUE053565T2 (enExample) |
| LT (1) | LT2844279T (enExample) |
| PL (1) | PL2844279T3 (enExample) |
| PT (1) | PT2844279T (enExample) |
| RS (1) | RS61598B1 (enExample) |
| SI (1) | SI2844279T1 (enExample) |
| SM (1) | SMT202100136T1 (enExample) |
| WO (1) | WO2013166249A1 (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10349884B2 (en) | 2011-06-03 | 2019-07-16 | Sighpath Pharma Inc. | Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel |
| US10117881B2 (en) | 2011-06-03 | 2018-11-06 | Signpath Pharma, Inc. | Protective effect of DMPC, DMPG, DMPC/DMPG, LYSOPG and LYSOPC against drugs that cause channelopathies |
| US10449193B2 (en) | 2011-06-03 | 2019-10-22 | Signpath Pharma Inc. | Protective effect of DMPC, DMPG, DMPC/DMPG, lysoPG and lysoPC against drugs that cause channelopathies |
| US12004868B2 (en) | 2011-06-03 | 2024-06-11 | Signpath Pharma Inc. | Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel |
| US10238602B2 (en) | 2011-06-03 | 2019-03-26 | Signpath Pharma, Inc. | Protective effect of DMPC, DMPG, DMPC/DMPG, LysoPG and LysoPC against drugs that cause channelopathies |
| WO2012167212A2 (en) | 2011-06-03 | 2012-12-06 | Signpath Pharma Inc. | Liposomal mitigation of drug-induced long qt syndrome and potassium delayed-rectifier current |
| EP2844279B1 (en) * | 2012-05-03 | 2020-12-09 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of pompe disease |
| WO2015095576A1 (en) * | 2013-12-18 | 2015-06-25 | Signpath Pharma, Inc. | Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel |
| US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| EP3207931A3 (en) | 2014-06-03 | 2017-12-20 | Signpath Pharma Inc. | Protective effect of dmpc, dmpg, dmpc/dmpg, egpg, lysopg and lysopc against drugs that cause channelopathies |
| TWI789758B (zh) * | 2014-09-30 | 2023-01-11 | 美商阿米庫斯醫療股份有限公司 | 高效經修飾之酸性α-葡萄糖苷酶及其製造與使用方法 |
| CN114540327B (zh) | 2014-09-30 | 2026-03-24 | 阿米库斯治疗学公司 | 具有增强的碳水化合物的高强度酸性α-葡糖苷酶 |
| KR102747683B1 (ko) | 2015-12-30 | 2024-12-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| RS64843B1 (sr) | 2015-12-30 | 2023-12-29 | Amicus Therapeutics Inc | Augmentirana kisela alfa-glukozidaza za lečenje pompeove bolesti |
| JP7046003B2 (ja) | 2016-03-30 | 2022-04-01 | アミカス セラピューティックス インコーポレイテッド | 高ｍ６ｐ組換えタンパク質の選択方法 |
| SG11201808455VA (en) | 2016-03-30 | 2018-10-30 | Amicus Therapeutics Inc | Formulations comprising recombinant acid alpha-glucosidase |
| KR102638618B1 (ko) | 2016-04-27 | 2024-02-21 | 사인패스 파마 인코포레이티드 | 약물 유발된 방실 차단의 방지 |
| WO2018209300A1 (en) * | 2017-05-12 | 2018-11-15 | Duke University | Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy |
| ES2950808T3 (es) | 2017-05-15 | 2023-10-13 | Amicus Therapeutics Inc | Alfa-glucosidasa ácida humana recombinante |
| WO2022227287A1 (zh) * | 2021-04-25 | 2022-11-03 | 中南大学 | Dnj及其衍生物在制备预防和/或治疗肺动脉高压药物中的应用 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6642038B1 (en) | 1999-09-14 | 2003-11-04 | Genzyme Glycobiology Research Institute, Inc. | GlcNAc phosphotransferase of the lysosomal targeting pathway |
| AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| EP1974752B1 (en) | 2001-04-30 | 2012-09-26 | BioMarin Pharmaceutical Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| WO2003086452A2 (en) | 2002-04-05 | 2003-10-23 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy |
| BRPI0411843B8 (pt) | 2003-06-24 | 2021-05-25 | Genzyme Corp | ácido nucleico compreendendo um promotor de beta-actina, vetor e método de produzir uma proteína |
| AU2005211775B2 (en) | 2004-02-06 | 2009-10-08 | Biomarin Pharmaceutical Inc. | Manufacture of highly phosphorylated lysosomal enzymes and uses thereof |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| AU2006247065B8 (en) * | 2005-05-17 | 2012-07-12 | Amicus Therapeutics, Inc. | A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2010056746A1 (en) * | 2008-11-11 | 2010-05-20 | Amicus Therapeutics, Inc. | Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease |
| EP2844279B1 (en) * | 2012-05-03 | 2020-12-09 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of pompe disease |
-
2013
- 2013-05-02 EP EP13785028.5A patent/EP2844279B1/en active Active
- 2013-05-02 EP EP24161179.7A patent/EP4397364A3/en active Pending
- 2013-05-02 JP JP2015510446A patent/JP6381521B2/ja active Active
- 2013-05-02 RS RS20210295A patent/RS61598B1/sr unknown
- 2013-05-02 SM SM20210136T patent/SMT202100136T1/it unknown
- 2013-05-02 ES ES20207542T patent/ES2980828T3/es active Active
- 2013-05-02 PL PL13785028T patent/PL2844279T3/pl unknown
- 2013-05-02 HU HUE13785028A patent/HUE053565T2/hu unknown
- 2013-05-02 WO PCT/US2013/039215 patent/WO2013166249A1/en not_active Ceased
- 2013-05-02 DK DK13785028.5T patent/DK2844279T3/da active
- 2013-05-02 EP EP20207542.0A patent/EP3871688B1/en active Active
- 2013-05-02 LT LTEP13785028.5T patent/LT2844279T/lt unknown
- 2013-05-02 ES ES13785028T patent/ES2859761T3/es active Active
- 2013-05-02 PT PT137850285T patent/PT2844279T/pt unknown
- 2013-05-02 US US14/398,210 patent/US20150086530A1/en active Pending
- 2013-05-02 HR HRP20210398TT patent/HRP20210398T1/hr unknown
- 2013-05-02 SI SI201331858T patent/SI2844279T1/sl unknown
-
2018
- 2018-01-19 JP JP2018007672A patent/JP6612370B2/ja active Active
-
2019
- 2019-10-29 JP JP2019196583A patent/JP6876111B2/ja active Active
-
2021
- 2021-01-27 JP JP2021010931A patent/JP7046241B2/ja active Active
- 2021-03-09 CY CY20211100202T patent/CY1124135T1/el unknown
-
2022
- 2022-03-22 JP JP2022044812A patent/JP2022101544A/ja not_active Withdrawn
-
2024
- 2024-03-07 JP JP2024034587A patent/JP2024081667A/ja active Pending
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