ATE442851T1 - 1-deoxy-galactonojirimycin-derivate zur vewendung bei der behandlung von lysosomalen speicherkrankheiten mittels steigerung der lysosomalen alpha galactosidase a - Google Patents

1-deoxy-galactonojirimycin-derivate zur vewendung bei der behandlung von lysosomalen speicherkrankheiten mittels steigerung der lysosomalen alpha galactosidase a

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Publication number
ATE442851T1
ATE442851T1 AT99925611T AT99925611T ATE442851T1 AT E442851 T1 ATE442851 T1 AT E442851T1 AT 99925611 T AT99925611 T AT 99925611T AT 99925611 T AT99925611 T AT 99925611T AT E442851 T1 ATE442851 T1 AT E442851T1
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AT
Austria
Prior art keywords
lysosomal
galactonojirimycin
deoxy
treatment
boosting
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Application number
AT99925611T
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English (en)
Inventor
Jian-Qiang Fan
Satoshi Ishii
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Sinai School Medicine
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Publication of ATE442851T1 publication Critical patent/ATE442851T1/de

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D451/00Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C07D451/06Oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pyrrole Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
AT99925611T 1998-06-01 1999-05-28 1-deoxy-galactonojirimycin-derivate zur vewendung bei der behandlung von lysosomalen speicherkrankheiten mittels steigerung der lysosomalen alpha galactosidase a ATE442851T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US09/087,804 US6274597B1 (en) 1998-06-01 1998-06-01 Method of enhancing lysosomal α-Galactosidase A
PCT/US1999/010479 WO1999062517A1 (en) 1998-06-01 1999-05-28 METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A

Publications (1)

Publication Number Publication Date
ATE442851T1 true ATE442851T1 (de) 2009-10-15

Family

ID=22207362

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99925611T ATE442851T1 (de) 1998-06-01 1999-05-28 1-deoxy-galactonojirimycin-derivate zur vewendung bei der behandlung von lysosomalen speicherkrankheiten mittels steigerung der lysosomalen alpha galactosidase a

Country Status (12)

Country Link
US (16) US6274597B1 (de)
EP (2) EP1083899B1 (de)
JP (2) JP4602548B2 (de)
AT (1) ATE442851T1 (de)
AU (1) AU4186199A (de)
CA (1) CA2333965C (de)
CY (2) CY1112845T1 (de)
DE (1) DE69941429D1 (de)
DK (2) DK1083899T3 (de)
ES (2) ES2507515T3 (de)
PT (2) PT2143420E (de)
WO (1) WO1999062517A1 (de)

Families Citing this family (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20050032841A1 (en) * 1999-04-20 2005-02-10 Steven Walkley Therapeutic compositions and methods of treating glycolipid storage related disorders
CA2398995C (en) * 2000-02-04 2014-09-23 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
DE60239000D1 (de) * 2001-09-07 2011-03-03 Seikagaku Kogyo Co Ltd Carbazuckeraminderivate und mittel gegen störungenff enthalten
US7842470B2 (en) * 2001-10-09 2010-11-30 Oregon Health & Science University Method for pharmacoperones correction of GnRHR mutant protein misfolding
WO2004004740A1 (en) * 2002-07-05 2004-01-15 King Roderick Frederick Gerard Aqueous formulation comprising digestible saccharides to reduce the tendency to cardiovascular disease in a subject
AU2003284886A1 (en) * 2002-10-21 2004-05-13 William E. Balch CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE
CN102586205A (zh) * 2003-01-31 2012-07-18 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US20070066543A1 (en) * 2003-05-22 2007-03-22 The Hospital For Sick Children Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity
FR2861991B1 (fr) 2003-11-07 2008-01-18 Centre Nat Rech Scient Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
BRPI0416216A (pt) * 2003-11-12 2006-12-26 Amicus Therapeutics Inc composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US7488721B2 (en) * 2004-05-13 2009-02-10 Mcmaster University Real time methylumbelliferone-based assay
US20090239807A1 (en) * 2004-05-24 2009-09-24 Mia Horowitz Methods and kits for diagnosing and/or assessing severity and treating gaucher disease
EP1773401B1 (de) * 2004-06-21 2013-01-02 Medtronic, Inc. Medizinische systeme und verfahren zur abgabe von zusammensetzungen an zellen
US8889127B2 (en) * 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
FR2873377B1 (fr) * 2004-07-23 2006-10-13 Sanofi Synthelabo Derives d'azasucre, inhibiteurs d'heparanases, leur procede de preparation, les compositions en contenant et leur utilisation
EP3782655A1 (de) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. Verfahren zur behandlung der pompe-krankheit unter verwendung von 1-deoxynojirimycin und derivaten
US20070021433A1 (en) * 2005-06-03 2007-01-25 Jian-Qiang Fan Pharmacological chaperones for treating obesity
WO2006133446A2 (en) * 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
FR2887549B1 (fr) 2005-06-23 2009-07-10 Centre Nat Rech Scient Nouveaux composes de la famille des iminosucres, leurs utilisations, notamment pour le traitement de maladies lysosomales, ainsi que leur procede de preparation
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2015230773B2 (en) * 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
EP2533050B3 (de) 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Behandlungsoptionen für Morbus Fabry
ES2364586T3 (es) * 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
DK1860101T3 (da) * 2006-05-24 2011-07-25 Amicus Therapeutics Inc Tartrat salt af isofagomin og fremgangsmåder til anvendelse deraf
JP5303458B2 (ja) 2006-06-23 2013-10-02 アミークス セラピューティックス インコーポレイテッド β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
KR20090040906A (ko) * 2006-08-02 2009-04-27 유나이티드 세러퓨틱스 코오포레이션 바이러스 감염의 리포솜 치료
US20100144008A1 (en) * 2006-12-21 2010-06-10 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of fabry disease
US20100184803A1 (en) * 2007-03-09 2010-07-22 Link Medicine Corporation Treatment of Lysosomal Storage Diseases
CA2682441A1 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
AU2008240138B2 (en) * 2007-04-13 2013-03-07 Amicus Therapeutics, Inc. Treatment of Gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
US20100197018A1 (en) * 2007-04-13 2010-08-05 Amicus Therapeutics, Inc. Use of substrates as pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
KR20100017879A (ko) 2007-05-22 2010-02-16 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
WO2009049421A1 (en) * 2007-10-18 2009-04-23 The Hospital For Sick Children Compositions and methods for enhancing enzyme activity in gaucher, gm1-gangliosidosis/morquio b disease, and parkinson's disease
WO2009066069A1 (en) * 2007-11-21 2009-05-28 Summit Corporation Plc Treatment of protein folding disorders
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
WO2009118658A2 (en) * 2008-03-26 2009-10-01 University Of Oxford Endoplasmic reticulum targeting liposomes
DK3578195T5 (da) 2008-06-26 2024-06-10 Zevra Denmark As Anvendelse af Hsp70 som regulator af enzymatisk aktivitet
WO2010048532A1 (en) * 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP2410989A2 (de) * 2009-03-27 2012-02-01 The Chancellor, Masters and Scholars of the University of Oxford Liposome zur senkung des cholesterinspiegels
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
JP5805630B2 (ja) * 2009-05-26 2015-11-04 アミカス セラピューティックス インコーポレイテッド 生物製剤の製造および精製を改善するための薬理学的シャペロンの利用
AR078498A1 (es) * 2009-10-01 2011-11-09 Baylor Res Inst Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry
KR101880411B1 (ko) * 2009-10-19 2018-07-19 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
US9044437B2 (en) 2009-10-19 2015-06-02 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
RU2608520C2 (ru) * 2009-10-19 2017-01-19 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения лизосомных болезней накопления
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
KR101784539B1 (ko) * 2010-01-28 2017-10-11 랩터 파마슈티컬스 인코포레이티드 수용체 결합 단백질(rap) 펩타이드-푸코시다제 억제제 접합체를 이용한 간 질환의 치료방법
CA2788983A1 (en) * 2010-02-02 2011-08-11 The Chancellor, Masters And Scholars Of The University Of Oxford Synthesis of dgjnac from d-glucuronolactone and use to inhibit alpha-n-acetylgalactosaminidases
WO2012072082A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
CN107088225A (zh) * 2011-03-11 2017-08-25 阿米库斯治疗学公司 治疗法布里病的给药方案
DK2753346T3 (da) 2011-09-07 2020-05-25 Sinai School Medicine Ceramidase og celledifferentiering
WO2013079580A1 (en) 2011-11-29 2013-06-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of darier disease
US9404100B2 (en) 2012-03-07 2016-08-02 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of Pompe disease
EP2854910B1 (de) 2012-06-01 2020-04-15 Icahn School of Medicine at Mount Sinai Ceramidspiegel bei der behandlung und vorbeugung von infektionen
ES2779006T3 (es) 2013-01-09 2020-08-13 Amicus Therapeutics Inc Composiciones de DNJ parenterales estables
EP3659619A1 (de) 2013-03-14 2020-06-03 Icahn School of Medicine at Mount Sinai Therapeutische säure-ceramidase-zusammensetzungen und verfahren zur herstellung und verwendung davon
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
HUE054957T2 (hu) 2014-09-15 2021-10-28 Orphazyme As Arimoklomol készítése
FI3201320T3 (fi) 2014-09-30 2024-01-08 Amicus Therapeutics Inc Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla
ES2978714T3 (es) 2015-02-05 2024-09-18 Cardiovalve Ltd Válvula protésica con marcos de deslizamiento axial
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA3010205A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
JP7098529B2 (ja) * 2016-03-22 2022-07-11 アミカス セラピューティックス インコーポレイテッド Gla遺伝子にg9331a変異を有する患者においてファブリー病を処置する方法
KR20250050123A (ko) 2016-03-30 2025-04-14 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
CA3287846A1 (en) 2016-04-29 2025-11-29 Zevra Denmark A/S Arimoclomol for treating glucocerebrosidase associated disorders
CA3031249A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
PT3624831T (pt) 2017-05-15 2023-07-04 Amicus Therapeutics Inc Alfa-glucosidase ácida humana recombinante
EP3630114B1 (de) 2017-05-30 2023-11-01 Amicus Therapeutics, Inc. Migalastat zur behandlung von nierenbeeinträchtigten patienten mit morbus fabry
KR102631805B1 (ko) 2017-05-30 2024-01-31 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
DK3630725T3 (da) 2017-06-01 2026-02-09 Idorsia Pharmaceuticals Ltd Krystallinsk form af n-butyldeoxygalactonojirimycin
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
SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
SMT202200459T1 (it) 2017-08-28 2023-01-13 Amicus Therapeutics Inc Metodi di potenziamento e/o stabilizzazione della funzione cardiaca in pazienti con malattia di fabry
WO2019051297A1 (en) * 2017-09-08 2019-03-14 The Nemours Foundation AGENT, DEVICE AND BLOOD CIRCULATION SYSTEM FOR TREATING LYSOSOMAL DISEASES, AND METHODS OF TREATING LYSOSOMAL DISEASES
US11318014B2 (en) 2017-09-19 2022-05-03 Cardiovalve Ltd. Prosthetic valve delivery system with multi-planar steering
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
ES2716305B2 (es) 2017-12-11 2019-11-27 Fund Biomedica Galicia Sur Uso de chaperonas farmacologicas para el tratamiento de enfermedades de deposito lisosomal
JP7555818B2 (ja) 2018-02-06 2024-09-25 アミカス セラピューティックス インコーポレイテッド 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
HUE064761T2 (hu) 2018-02-06 2024-04-28 Amicus Therapeutics Inc Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal
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
US20210315875A1 (en) 2018-08-20 2021-10-14 Amicus Therapeutics, Inc. Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene
TW202042812A (zh) 2019-01-22 2020-12-01 美商阿米庫斯醫療股份有限公司 減少法布瑞氏症患者中之腦血管事件之方法
US20220313670A1 (en) 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
HRP20260109T1 (hr) 2019-08-07 2026-03-13 Amicus Therapeutics, Inc. Migalastat za upotrebu u liječenju fabryjeve bolesti kod pacijenata koji imaju mutaciju u gla genu
US20220273615A1 (en) * 2019-10-04 2022-09-01 Academia Sinica Methods of treating pompe disease
AU2020391220A1 (en) * 2019-11-27 2022-06-30 Turning Point Therapeutics, Inc. Combination therapy involving diaryl macrocyclic compounds
US20210251972A1 (en) * 2020-02-10 2021-08-19 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease
CA3170718A1 (en) 2020-03-06 2021-09-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
FR3109523B1 (fr) * 2020-04-23 2023-11-17 Univ Grenoble Alpes Nouveaux composés chaperons pharmacologiques de l’alpha-glucosidase acide humaine et leur utilisation thérapeutique
KR20230128462A (ko) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
EP4370120A1 (de) 2021-07-12 2024-05-22 Amicus Therapeutics, Inc. Verfahren zur behandlung von morbus fabry bei pädiatrischen patienten
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
US20240197706A1 (en) 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NO154918C (no) 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
JPS61280472A (ja) 1985-06-05 1986-12-11 Shionogi & Co Ltd β−ガラクトシダ−ゼ阻害物質ガラクトスタチン類およびその製造方法
ATE126696T1 (de) 1985-12-23 1995-09-15 Hutchinson Fred Cancer Res Regulierung der retroviralen replikation, infektion und pathogenese.
DE3737523A1 (de) 1987-11-05 1989-05-18 Bayer Ag Verwendung von substituierten hydroxypiperidinen als antivirale mittel
US5192772A (en) 1987-12-09 1993-03-09 Nippon Shinyaku Co. Ltd. Therapeutic agents
US5250545A (en) 1988-02-12 1993-10-05 Meiji Seika Kaisha, Ltd. Cancer cell metastasis inhibitor methods
FR2640621B1 (fr) 1988-12-19 1992-10-30 Centre Nat Rech Scient N-aryl-azetidinones, leur procede de preparation et leur utilisation comme inhibiteurs des elastases
US5043273A (en) 1989-08-17 1991-08-27 Monsanto Company Phosphorylated glycosidase inhibitor prodrugs
US5030638A (en) 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US5504078A (en) 1990-06-08 1996-04-02 Merrell Dow Pharmaceuticals Inc. α-glucosidase inhibitors
WO1992000277A1 (en) 1990-06-29 1992-01-09 Nippon Shinyaku Co., Ltd. Piperidine derivative
US5276120A (en) 1991-05-30 1994-01-04 The Scripps Research Institute Process for forming omega-deuxy-azasugars
US5399567A (en) * 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US6291657B1 (en) * 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
JPH0748398A (ja) 1993-08-03 1995-02-21 Nippon Oil Co Ltd 変性蛋白質の再生方法及び変性蛋白質再生剤
US5691306A (en) 1993-08-26 1997-11-25 National Research Council Of Canada Methods of detection and treatment of protein trafficking disorders and increasing secretory protein production
JP3628021B2 (ja) 1994-01-13 2005-03-09 ジー.ディー.サール アンド カンパニー B型肝炎ウイルス感染症の治療のための1,5−ジデオキシ−1,5−イミノ−d−グルシトールのn−アルキル誘導体の使用
WO1995022975A1 (en) 1994-02-25 1995-08-31 G.D. Searle & Co. Use of 1-deoxynojirimycin and its derivatives for treating mammals infected with respiratory syncytial virus
US5863903A (en) 1994-03-09 1999-01-26 Novo Nordisk A/S Use of hydroxy alkyl piperidine and pyrrolidine compounds to treat diabetes
US5844102A (en) 1994-07-07 1998-12-01 University Of Maryland Baltimore County Glycohydrolase inhibitors, their preparation and use thereof
WO1998002161A1 (en) 1996-07-15 1998-01-22 Universiteit Van Amsterdam Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
HU227189B1 (en) 1996-09-13 2010-10-28 Transkaryotic Therapies Therapy for alpha-galactosidase a deficiency
GB9700233D0 (en) 1997-01-08 1997-02-26 Univ London Use of glycosidase inhibitors
US6210666B1 (en) * 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
ZA9810210B (en) 1997-11-10 1999-07-14 Searle & Co Use of alkylated iminosugars to treat multidrug resistance.
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
WO1999040435A1 (en) 1998-02-09 1999-08-12 Netzer William J Protein folding inhibitors
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
WO2000029556A2 (en) 1998-11-17 2000-05-25 The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services Identification of glycosphingolipids that promote hiv-1 entry into cells
TR200101549T2 (tr) 1998-12-02 2001-11-21 Pfizer Products Inc. p53-Familyasından bir proteinin yapısal kararlılığının canlandırılmasına yönelik metotlar.
US6610703B1 (en) 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
JP2000219632A (ja) 1999-01-28 2000-08-08 Toyotama Koryo Kk カロリー軽減剤
WO2000056334A1 (en) 1999-03-19 2000-09-28 The Trustees Of Boston College Use of imino sugars for anti-tumor therapy
AR023541A1 (es) 1999-04-19 2002-09-04 Schering Corp Terapia de combinacion para vhc
GB9909064D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
US6476194B1 (en) 1999-06-29 2002-11-05 National Research Council Of Canada Method for folding unfolded proteins
AU6050600A (en) 1999-07-26 2001-02-13 G.D. Searle & Co. Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidaseenzyme for the manufacture of medicament for the treatment of glycolipid storag e diseases
HK1046869A1 (zh) 1999-08-10 2003-01-30 The Chancellor, Masters And Scholars Of The University Of Oxford 使用长链n烷基化合物及其恶衍生物作为抗病毒成分
AU7712000A (en) 1999-09-24 2001-04-24 Johns Hopkins University School Of Medicine, The Methods for identifying an agent that corrects defective protein folding
US6656912B2 (en) 2000-01-20 2003-12-02 Washington University In St. Louis Methods to treat α1-antitrypsin deficiency
WO2001097829A2 (en) 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
WO2002028348A2 (en) 2000-10-04 2002-04-11 The Children's Hospital Of Philadelphia Compositions and methods for treatment of cystic fibrosis
JP2002190202A (ja) 2000-10-12 2002-07-05 Koito Mfg Co Ltd ヘッドランプ
JP3589449B2 (ja) 2001-02-26 2004-11-17 株式会社ユニレック 静電容量センサ、管内流動判断装置、管内流動制御装置
JP4802548B2 (ja) * 2005-04-28 2011-10-26 シンフォニアテクノロジー株式会社 Icチップ実装体の製造装置
US7174582B1 (en) * 2005-08-17 2007-02-13 Kevin J Leary Crib mattress bouncer
US20110053901A1 (en) 2008-02-14 2011-03-03 Signum Biosciences, Inc. Acetyl mimic compounds for the inhibition of isoprenyl-s-cysteinyl methyltransferase
US20100247461A1 (en) 2009-01-20 2010-09-30 Signum Biosciences, Inc. Anti-inflammatory complexes
CN102209701B (zh) 2008-11-11 2014-10-15 西格纳姆生物科学公司 异戊二烯基化合物和其方法

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US20030119874A1 (en) 2003-06-26
ES2333561T3 (es) 2010-02-23
EP2143420A1 (de) 2010-01-13
US7141582B2 (en) 2006-11-28
JP5528941B2 (ja) 2014-06-25
JP4602548B2 (ja) 2010-12-22
CA2333965A1 (en) 1999-12-09
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US6274597B1 (en) 2001-08-14
US20100137229A1 (en) 2010-06-03
US8841322B2 (en) 2014-09-23
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US20040242539A1 (en) 2004-12-02
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US8633221B2 (en) 2014-01-21
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