KR102076585B1 - 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 - Google Patents

리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 Download PDF

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KR102076585B1
KR102076585B1 KR1020147019849A KR20147019849A KR102076585B1 KR 102076585 B1 KR102076585 B1 KR 102076585B1 KR 1020147019849 A KR1020147019849 A KR 1020147019849A KR 20147019849 A KR20147019849 A KR 20147019849A KR 102076585 B1 KR102076585 B1 KR 102076585B1
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KR20140107481A (ko
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얀 루카스
아나히트 퓨스-다브티안
마티아스 벨러
아른트 롤프스
샤리프 무함마드
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센토제너 아이피 게엠베하
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/137—Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Emergency Medicine (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Hydrogenated Pyridines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020147019849A 2011-12-22 2012-12-21 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 Expired - Fee Related KR102076585B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP11010078.1 2011-12-22
EP11010078 2011-12-22
PCT/EP2012/005363 WO2013091897A1 (en) 2011-12-22 2012-12-21 Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol

Publications (2)

Publication Number Publication Date
KR20140107481A KR20140107481A (ko) 2014-09-04
KR102076585B1 true KR102076585B1 (ko) 2020-02-12

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KR1020147019849A Expired - Fee Related KR102076585B1 (ko) 2011-12-22 2012-12-21 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물

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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=)

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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

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WO2007137072A2 (en) 2006-05-16 2007-11-29 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
US20110189710A1 (en) 2008-03-12 2011-08-04 Amicus Therapeutics, Inc. TREATMENT OF POMPE DISEASE WITH SPECIFIC PHARMACOLOGICAL CHAPERONES AND MONITORING TREATMENT USING SURROGATE MARkERS

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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
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
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

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
WO2007137072A2 (en) 2006-05-16 2007-11-29 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
US20110189710A1 (en) 2008-03-12 2011-08-04 Amicus Therapeutics, Inc. TREATMENT OF POMPE DISEASE WITH SPECIFIC PHARMACOLOGICAL CHAPERONES AND MONITORING TREATMENT USING SURROGATE MARkERS

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US20210228560A1 (en) 2021-07-29
AU2012359165A1 (en) 2014-07-10
EP2793872B1 (en) 2021-03-10
AU2017279780A1 (en) 2018-01-25
ES2864774T3 (es) 2021-10-14
IL233173A0 (en) 2014-07-31
KR20140107481A (ko) 2014-09-04
AU2017279780B2 (en) 2019-10-03
EP2793872A1 (en) 2014-10-29
WO2013091897A8 (en) 2014-10-09
BR112014014808A2 (pt) 2018-09-25
JP2015502964A (ja) 2015-01-29
IL233173B (en) 2020-10-29
CA2860085A1 (en) 2013-06-27
JP2018076353A (ja) 2018-05-17
JP6320931B2 (ja) 2018-05-09
WO2013091897A1 (en) 2013-06-27
US20150258081A1 (en) 2015-09-17

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