JP5337405B2 - ゴーシェ病の治療方法 - Google Patents

ゴーシェ病の治療方法 Download PDF

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Publication number
JP5337405B2
JP5337405B2 JP2008135368A JP2008135368A JP5337405B2 JP 5337405 B2 JP5337405 B2 JP 5337405B2 JP 2008135368 A JP2008135368 A JP 2008135368A JP 2008135368 A JP2008135368 A JP 2008135368A JP 5337405 B2 JP5337405 B2 JP 5337405B2
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gcase
ambroxol
acid
pharmaceutically acceptable
activity
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JP2008135368A
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Japanese (ja)
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JP2009137933A (ja
JP2009137933A5 (https=
Inventor
ドン・ジェイ・マフラン
マイケル・ビー・トロパック
ジャスティン・ディー・バトナー
ジャン・イー・ブランチャード
エリック・ディー・ブラウン
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McMaster University
Hospital for Sick Children HSC
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McMaster University
Hospital for Sick Children HSC
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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
    • A61K31/13—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/136—Amines having aromatic rings, e.g. ketamine, nortriptyline having the amino group directly attached to the aromatic ring, e.g. benzeneamine
    • 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
    • A61K31/138—Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
    • 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
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
    • A61K31/167—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the nitrogen of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
    • 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
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/235—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids having an aromatic ring attached to a carboxyl group
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • 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
    • A61K31/54—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame
    • A61K31/5415—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame ortho- or peri-condensed with carbocyclic ring systems, e.g. phenothiazine, chlorpromazine, piroxicam
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/553—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and one oxygen as ring hetero atoms, e.g. loxapine, staurosporine
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Emergency Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2008135368A 2007-09-17 2008-05-23 ゴーシェ病の治療方法 Active JP5337405B2 (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US97296807P 2007-09-17 2007-09-17
US60/972,968 2007-09-17
US6555008P 2008-02-12 2008-02-12
US61/065,550 2008-02-12
US6568408P 2008-02-13 2008-02-13
US61/065,684 2008-02-13

Publications (3)

Publication Number Publication Date
JP2009137933A JP2009137933A (ja) 2009-06-25
JP2009137933A5 JP2009137933A5 (https=) 2011-06-16
JP5337405B2 true JP5337405B2 (ja) 2013-11-06

Family

ID=39875224

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2008135368A Active JP5337405B2 (ja) 2007-09-17 2008-05-23 ゴーシェ病の治療方法

Country Status (6)

Country Link
US (10) US8124597B2 (https=)
EP (1) EP2200596B1 (https=)
JP (1) JP5337405B2 (https=)
CA (1) CA2699864C (https=)
IL (1) IL204285A (https=)
WO (1) WO2009038695A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102004021992A1 (de) * 2004-05-03 2005-11-24 Boehringer Ingelheim Pharma Gmbh & Co. Kg Topische Zubereitung enthaltend Ambroxol
EP3366688B1 (en) 2010-12-08 2022-02-09 The U.S.A. as represented by the Secretary, Department of Health and Human Services Substituted pyrazolopyrimidines as glucocerebrosidase activators
KR102076585B1 (ko) * 2011-12-22 2020-02-12 센토제너 아이피 게엠베하 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물
KR102227473B1 (ko) 2013-04-15 2021-03-15 삼성디스플레이 주식회사 박막 증착 장치 및 이를 이용한 박막 형성 방법
CN106455661A (zh) * 2014-05-21 2017-02-22 雀巢产品技术援助有限公司 营养物质的个性化补充
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
CN110325516A (zh) 2016-12-29 2019-10-11 米尼奥尔克斯治疗有限公司 杂芳基化合物和它们的用途
JP7369716B2 (ja) * 2018-01-26 2023-10-26 ノイレ， エルエルシー 皮膚バリア機能を改善するためのアンブロキソールの使用
WO2019211205A1 (en) 2018-04-30 2019-11-07 Dorphan S.A. Pharmaceutical compositions for use in the treatment of lysosomal diseases
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
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
WO2025153714A1 (en) * 2024-01-19 2025-07-24 Scenic Biotech Bv Pla2g15 inhibitors for use in the treatment of lysosomal storage diseases, hiv, alzheimer's disease or parkinson's disease
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ID28460A (id) 1998-07-08 2001-05-24 Lipogenics Inc Komposisi dan metoda untuk perlakuan dan pencegahan penyakit tulang dengan menggunakan tocotrienol
MXPA04002066A (es) * 2001-09-04 2005-02-17 Techno Network Shinoku Co Ltd Farmacos contra influenza.
US20030171391A1 (en) * 2002-01-25 2003-09-11 Boehringer Ingelheim Pharma Gmbh & Co. Kg Ambroxol for the treatment of chronic pain
DE10303229B4 (de) * 2003-01-28 2007-07-26 Keyneurotek Ag Ambroxol und Inhibitoren des Angiotensin Converting-Enzyms (ACE) umfassende Arzneimittel Arzneimittel-Zubereitung und ihre Verwendung zur Behandlung von neurodegenerativen Erkrankungen
DE10332472A1 (de) * 2003-07-16 2005-02-03 Boehringer Ingelheim Pharma Gmbh & Co. Kg Ambroxol für die Behandlung von Tinnitus
US7488721B2 (en) * 2004-05-13 2009-02-10 Mcmaster University Real time methylumbelliferone-based assay
EP1850840A2 (en) * 2005-01-13 2007-11-07 Sirtris Pharmaceuticals, Inc. Novel compositions for preventing and treating neurodegenerative and blood coagulation disorders
JP2008528510A (ja) * 2005-01-20 2008-07-31 サートリス ファーマシューティカルズ， インコーポレイテッド 紅潮および／または薬物誘発性体重増加を処置するためのサーチュイン活性化化合物の使用
US20070149466A1 (en) * 2005-07-07 2007-06-28 Michael Milburn Methods and related compositions for treating or preventing obesity, insulin resistance disorders, and mitochondrial-associated disorders
US8795668B2 (en) * 2005-12-23 2014-08-05 The Regents Of The University Of Michigan Methods for treating pulmonary fibrosis
US20070244184A1 (en) * 2006-01-09 2007-10-18 Simon Fraser University Glycosidase inhibitors and methods of synthesizing same

Also Published As

Publication number Publication date
US8937059B2 (en) 2015-01-20
US11160773B2 (en) 2021-11-02
EP2200596A1 (en) 2010-06-30
IL204285A (en) 2013-10-31
US20160113892A1 (en) 2016-04-28
EP2200596B1 (en) 2012-06-13
US20130090388A1 (en) 2013-04-11
US9415025B2 (en) 2016-08-16
JP2009137933A (ja) 2009-06-25
US9006223B2 (en) 2015-04-14
CA2699864A1 (en) 2009-03-26
US20160331701A1 (en) 2016-11-17
CA2699864C (en) 2016-08-02
US20090075960A1 (en) 2009-03-19
US20180296502A1 (en) 2018-10-18
WO2009038695A1 (en) 2009-03-26
US9233083B2 (en) 2016-01-12
US20130184352A1 (en) 2013-07-18
US10028922B2 (en) 2018-07-24
US8124597B2 (en) 2012-02-28
US20140235713A1 (en) 2014-08-21
US20200246282A1 (en) 2020-08-06
US10653645B2 (en) 2020-05-19
US8951994B2 (en) 2015-02-10
US20140249233A1 (en) 2014-09-04
US20120021487A1 (en) 2012-01-26
US8404668B2 (en) 2013-03-26

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