JP2018076353A5 - - Google Patents

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Publication number
JP2018076353A5
JP2018076353A5 JP2017248953A JP2017248953A JP2018076353A5 JP 2018076353 A5 JP2018076353 A5 JP 2018076353A5 JP 2017248953 A JP2017248953 A JP 2017248953A JP 2017248953 A JP2017248953 A JP 2017248953A JP 2018076353 A5 JP2018076353 A5 JP 2018076353A5
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JP
Japan
Prior art keywords
pharmaceutically acceptable
acceptable salt
formula
dideoxy
acetamido
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
Application number
JP2017248953A
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English (en)
Japanese (ja)
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JP2018076353A (ja
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Publication date
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Publication of JP2018076353A publication Critical patent/JP2018076353A/ja
Publication of JP2018076353A5 publication Critical patent/JP2018076353A5/ja
Pending legal-status Critical Current

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JP2017248953A 2011-12-22 2017-12-26 リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび/またはアンブロキソールの誘導体の組み合わせ Pending JP2018076353A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP11010078.1 2011-12-22
EP11010078 2011-12-22

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2014547767A Division JP6320931B2 (ja) 2011-12-22 2012-12-21 リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび/またはアンブロキソールの誘導体の組み合わせ

Publications (2)

Publication Number Publication Date
JP2018076353A JP2018076353A (ja) 2018-05-17
JP2018076353A5 true JP2018076353A5 (https=) 2018-07-19

Family

ID=47559375

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2014547767A Expired - Fee Related JP6320931B2 (ja) 2011-12-22 2012-12-21 リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび/またはアンブロキソールの誘導体の組み合わせ
JP2017248953A Pending JP2018076353A (ja) 2011-12-22 2017-12-26 リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび/またはアンブロキソールの誘導体の組み合わせ

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2014547767A Expired - Fee Related JP6320931B2 (ja) 2011-12-22 2012-12-21 リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび/またはアンブロキソールの誘導体の組み合わせ

Country Status (10)

Country Link
US (2) US20150258081A1 (https=)
EP (1) EP2793872B1 (https=)
JP (2) JP6320931B2 (https=)
KR (1) KR102076585B1 (https=)
AU (2) AU2012359165A1 (https=)
BR (1) BR112014014808A2 (https=)
CA (1) CA2860085A1 (https=)
ES (1) ES2864774T3 (https=)
IL (1) IL233173B (https=)
WO (1) WO2013091897A1 (https=)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
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
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP6981961B2 (ja) * 2015-07-30 2021-12-17 ホライズン オルファン エルエルシー フコシダーゼ阻害剤
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
KR20190087673A (ko) 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US20190254992A1 (en) * 2016-06-29 2019-08-22 Hadasit Medical Research Services & Development Limited Combinations of beta-glycolipides and 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol, compositions and uses thereof in the treatment of disorders associated with protein misfolding and protein aggregations
WO2018089797A1 (en) * 2016-11-14 2018-05-17 Mingwu Wang Formulations for the treatment of ocular surface diseases and related methods
CN110573492A (zh) * 2017-04-25 2019-12-13 阿米库斯治疗学公司 用于预防和/或治疗中枢神经系统退行性失调和/或溶酶体贮积失调的新颖组合物
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry 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
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
US11612619B2 (en) 2018-11-01 2023-03-28 National Institute Of Health (Nih), U.S. Dept. Of Health And Human Services (Dhhs), U.S. Government Compostions and methods for enabling cholesterol catabolism in human cells
EP3884383B1 (en) * 2018-12-21 2025-04-16 Shenzhen Yinwang Intelligent Technologies Co., Ltd. Data deterministic deliverable communication technology based on qos as a service
WO2020168294A1 (en) * 2019-02-15 2020-08-20 Lysosomal And Rare Disorders Research And Treatment Center, Llc Methods and compositions for treating lysosomal storage disorders: ambroxol as treatment agent for mucopolysaccharidoses iii (sanfilippo syndrome)
EP3996696A1 (en) * 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
US12544402B2 (en) 2020-02-28 2026-02-10 Repair Biotechnologies, Inc. Targeted expression of microbial cholesterol catalysis genes reduces excess lipid
CN112773779B (zh) * 2021-02-04 2022-08-16 上海交通大学医学院附属新华医院 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用
CN114644572B (zh) * 2022-03-22 2023-03-28 华南农业大学 一种溴己新半抗原、人工抗原、抗体及其制备方法和应用
KR20260002803A (ko) * 2023-05-02 2026-01-06 지위, 엘엘씨 치료적 사용을 위한 암브록솔의 변형된 형태

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19933148A1 (de) 1999-07-20 2001-01-25 Boehringer Ingelheim Int Ambroxolhaltige Lutschtablette
EP2857036B1 (en) * 2003-01-31 2016-12-21 Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
PL2533050T6 (pl) * 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
JP5337405B2 (ja) * 2007-09-17 2013-11-06 ザ・ホスピタル・フォー・シック・チルドレン ゴーシェ病の治療方法
CA2718182A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
EP2353403B1 (de) * 2010-02-01 2012-07-11 Symrise AG Verwendung von 1-(2,4-Dihydroxy-phenyl)-3-(3-hydroxy-4-methoxy-phenyl)-propan-1-on

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