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

Info

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
Authority
AT
Austria
Prior art keywords
lysosomal
galactonojirimycin
deoxy
treatment
boosting
Prior art date
Application number
AT99925611T
Other languages
English (en)
Inventor
Jian-Qiang Fan
Satoshi Ishii
Original Assignee
Sinai School Medicine
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sinai School Medicine filed Critical Sinai School Medicine
Application granted granted Critical
Publication of ATE442851T1 publication Critical patent/ATE442851T1/de

Links

Classifications

    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Hydrogenated Pyridines (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) EP2143420B1 (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) DK2143420T3 (de)
ES (2) ES2507515T3 (de)
PT (2) PT1083899E (de)
WO (1) WO1999062517A1 (de)

Families Citing this family (103)

* 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
EP2204360A1 (de) * 2001-09-07 2010-07-07 Seikagaku Corporation Carba-Zuckeramin-Derivat und Glycolipid-Stoffwechselerkrankungs-Behandlungsmittel mit den gleichen Inhaltsstoffen wie der aktive Inhaltsstoff
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
WO2004037373A2 (en) * 2002-10-21 2004-05-06 The Scripps Research Institute CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE
EP2444102B1 (de) * 2003-01-31 2015-04-08 The Mount Sinai School of Medicine of New York University Kombinationstherapie zur Behandlung von Proteinmangelstörungen
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
WO2004103368A1 (en) * 2003-05-22 2004-12-02 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
ES2402182T3 (es) * 2003-11-12 2013-04-29 Amicus Therapeutics Inc. Derivados de hidroxipiperidina para tratar la enfermedad de Gaucher
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
JP2008503590A (ja) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド 組成物を細胞にデリバリーするための医学用システム及び方法
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
WO2006125141A2 (en) 2005-05-17 2006-11-23 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives
US20070021433A1 (en) 2005-06-03 2007-01-25 Jian-Qiang Fan Pharmacological chaperones for treating obesity
EP1888068B1 (de) 2005-06-08 2014-05-21 Amicus Therapeutics, Inc. Behandlung von zns-erkrankungen in zusammenhang mit mutationen bei für lysosomale enzyme kodierenden genen
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
SI2533050T1 (sl) 2006-05-16 2014-07-31 Amicus Therapeutics, Inc. MoĹľnosti zdravljenja Fabrijeve bolezni
AU2015230773B2 (en) * 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
PT1860101E (pt) * 2006-05-24 2011-07-12 Amicus Therapeutics Inc Sal de tartrato da isofagomina e métodos de uso
ES2364586T3 (es) * 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
AU2007260812B2 (en) 2006-06-23 2012-08-16 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase
CA2659858A1 (en) * 2006-08-02 2008-07-24 United Therapeutics Corporation Liposome treatment of viral infections
EP2121005A1 (de) * 2006-12-21 2009-11-25 Academisch Ziekenhuis bij de Universiteit van Amsterdam Behandlung von morbus fabry
WO2008112525A2 (en) * 2007-03-09 2008-09-18 Link Medicine Corporation Treatment of lysosomal storage diseases
EP2142197A4 (de) * 2007-03-30 2010-11-10 Amicus Therapeutics Inc Verfahren zur behandlung von morbus fabry mit pharmakologischen chaperonen
JP5586453B2 (ja) * 2007-04-13 2014-09-10 アミカス セラピューティックス インコーポレイテッド 特異的薬理シャペロンによるゴーシェ病の治療および代理マーカーを用いた治療の監視
EP2137204A4 (de) * 2007-04-13 2010-05-12 Amicus Therapeutics Inc Verwendung von substraten als pharmakologische chaperone
JP2010525084A (ja) * 2007-04-26 2010-07-22 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
BRPI0812381A2 (pt) 2007-05-22 2016-08-09 Amicus Therapeutics Inc novo método para preparar isofagomina e seus derivados
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
EP2252288A1 (de) * 2007-11-21 2010-11-24 Summit Corporation Plc Behandlung von proteinfaltungsstörungen
HUE051377T2 (hu) 2008-02-12 2021-03-01 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
EP2282723A2 (de) * 2008-03-26 2011-02-16 University of Oxford Liposome mit endoplasmischem retikulum als target
ES2963910T3 (es) 2008-06-26 2024-04-03 Zevra Denmark As Uso de Hsp70 como regulador de la actividad enzimática
US8321148B2 (en) * 2008-10-24 2012-11-27 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
CA2763464C (en) * 2009-05-26 2018-11-06 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
WO2011041718A2 (en) * 2009-10-01 2011-04-07 Baylor Research Institute Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
RU2016103139A (ru) 2009-10-19 2018-11-22 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения лизосомных болезней накопления
WO2011049736A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating degenerative disorders of the central nervous system
ES2549504T3 (es) 2009-10-19 2015-10-28 Amicus Therapeutics, Inc. Método para el tratamiento de la enfermedad de Alzheimer utilizando chaperonas farmacológicas para aumentar la actividad de gangliosidasas
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
EP2528613B1 (de) * 2010-01-28 2015-10-21 Raptor Pharmaceuticals Inc. Peptid-fucosidase-hemmerkonjugaten aus einem rezeptor-assoziierten protein und deren verwendung zur behandlung von lebertumoren
KR20130009751A (ko) * 2010-02-02 2013-01-23 더 챈슬러 마스터즈 앤드 스칼라스 오브 더 유니버시티 오브 옥스포드 D-글루쿠로노락톤으로부터 DGJNAc의 합성 및 알파-N-아세틸갈락토사미니다제를 억제하기 위한 용도
US9662375B2 (en) 2010-11-30 2017-05-30 Orphazyme Aps Methods for increasing intracellular activity of Hsp70
JP2014528901A (ja) * 2011-03-11 2014-10-30 アミカス セラピューティックス インコーポレイテッド ファブリー病の治療用投薬レジメン
PL2753346T3 (pl) 2011-09-07 2020-10-19 Mount Sinai School Of Medicine Ceramidaza i różnicowanie komórek
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
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
WO2013181530A1 (en) 2012-06-01 2013-12-05 Icahn School Of Medicine At Mount Sinai Ceramide levels in the treatment and prevention of infections
US9827189B2 (en) 2013-01-09 2017-11-28 Amicus Therapeautics, Inc. Stable parenteral DNJ compositions
MX2015012471A (es) 2013-03-14 2016-08-03 Icahn School Med Mount Sinai Composiciones terapéuticas de ceramidasa ácida y métodos para prepararlas y utilizarlas.
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
RU2745292C2 (ru) 2014-09-15 2021-03-23 Орпхазиме А/C Состав с аримокломолом
CA3228032A1 (en) 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
CA2973940C (en) 2015-02-05 2022-08-23 Mitraltech Ltd. Prosthetic valve with axially-sliding frames
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2017117407A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
EP3432882B1 (de) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Behandlung von morbus fabry in patienten mit der mutation g9331a im gla gen
IL301319A (en) 2016-03-30 2023-05-01 Amicus Therapeutics Inc Formulations that include recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
EP3448382B1 (de) 2016-04-29 2020-10-14 Orphazyme A/S Arimoclomol zur behandlung mit glucocerebrosidase assoziierten störungen
EP4378524A3 (de) 2016-07-19 2024-08-14 Amicus Therapeutics, Inc. Behandlung von morbus fabry bei ert-naiven und ert-erfahrenen patienten
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
KR102631805B1 (ko) 2017-05-30 2024-01-31 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
TW202322814A (zh) 2017-05-30 2023-06-16 美商阿米庫斯醫療股份有限公司 治療有腎損傷的法布里患者的方法
TWI770184B (zh) 2017-06-01 2022-07-11 瑞士商愛杜西亞製藥有限公司 N-丁基脫氧半乳糖野尻黴素(N-butyldeoxygalactonojirimycin)之結晶型
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
WO2019017938A1 (en) 2017-07-19 2019-01-24 Amicus Therapeutics, Inc. TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
US11819405B2 (en) 2017-09-19 2023-11-21 Cardiovalve Ltd. Prosthetic valve with inflatable cuff configured for radial extension
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
JP7252211B2 (ja) * 2017-09-08 2023-04-04 ザ ヌムール ファウンデーション リソソーム蓄積症を治療するための薬剤、デバイス、および血液循環システム、ならびにリソソーム蓄積症を治療するための方法
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
US11357784B2 (en) 2018-02-06 2022-06-14 Amicus Therapeutics, Inc. Use of migalastat for treating Fabry disease in pregnant patients
AR114392A1 (es) 2018-02-06 2020-09-02 Amicus Therapeutics Inc Tratamiento de pacientes con enfermedad de fabry clásica
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
EP4299117A3 (de) 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Verfahren zur behandlung von morbus fabry bei patienten mit einer mutation in dem gla-gen
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
CA3141226A1 (en) 2019-06-11 2020-12-17 Franklin Johnson Methods of treating fabry disease in patients having renal impairment
EP3851105A3 (de) 2019-08-07 2021-10-13 Amicus Therapeutics, Inc. Verfahren zur behandlung von morbus fabry bei patienten mit einer mutation in dem gla-gen
WO2021067324A1 (en) 2019-10-04 2021-04-08 Academia Sinica Methods of treating pompe disease
CA3163095A1 (en) * 2019-11-27 2021-06-03 Turning Point Therapeutics, Inc. Combination therapy involving diaryl macrocyclic compounds
AU2021218578A1 (en) * 2020-02-10 2022-08-25 Amicus Therapeutics, Inc. Methods of treating Fabry disease
BR112022017881A2 (pt) 2020-03-06 2022-12-06 Amicus Therapeutics Inc Métodos de tratamento da doença de fabry em pacientes que têm uma mutação no gene gla
JP2024500632A (ja) 2020-11-19 2024-01-10 ゼブラ デンマーク エー/エス アリモクロモルクエン酸塩及びその中間体の調製プロセス
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
JP2024525760A (ja) 2021-07-12 2024-07-12 アミカス セラピューティックス インコーポレイテッド 小児患者のファブリー病を治療する方法
EP4413376A1 (de) 2021-10-08 2024-08-14 Amicus Therapeutics, Inc. Biomarker für lysosomale speicherkrankheiten
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
JPS63501925A (ja) 1985-12-23 1988-08-04 フレツド ハツチンソン キヤンサ− リサ−チ センタ− レトロウイルスの複製、感染及び疾病誘発の制御
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 (fr) 1990-06-29 1992-01-09 Nippon Shinyaku Co., Ltd. Derive de piperidine
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
AU1876095A (en) 1994-02-25 1995-09-11 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
EP0912179B1 (de) 1996-07-15 2006-04-12 MacroZyme DNM B.V. Deoxynojirimycin derivate und ihre verwendung als glukosesylceramidase-inhibitoren
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
JP2001522833A (ja) 1997-11-10 2001-11-20 ジー・ディー・サール・アンド・カンパニー 多剤耐性を治療するためのアルキル化イミノ糖類の使用
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
AU2590099A (en) 1998-02-09 1999-08-23 William J. Netzer 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
AU1734600A (en) 1998-11-17 2000-06-05 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Identification of glycosphingolipids that promote hiv-1 entry into cells
ID29061A (id) 1998-12-02 2001-07-26 Pfizer Prod Inc METODE DAN KOMPOSISI UNTUK MEMPERBAIKI STABILITAS KONFORMASI SUATU PROTEIN DARI KELUARGA p53
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
CO5241354A1 (es) 1999-04-19 2003-01-31 Schering Corp Terapia de combinacio 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
CA2378776A1 (en) 1999-07-26 2001-02-01 G.D. Searle & Co. Use of long-chain n-alkyl derivatives of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of medicament for the treatment of glycolipid storage diseases
WO2001010429A2 (en) 1999-08-10 2001-02-15 The Chancellor, Masters, And Scholars Of The University Of Oxford Long chain n-alkyl compounds and oxa-derivatives thereof and use as antiviral compositions
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
WO2001052830A2 (en) 2000-01-20 2001-07-26 Washington University METHODS TO TREAT α-1-ANTITRYPSIN DEFICIENCY
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker 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
AU2009214493A1 (en) 2008-02-14 2009-08-20 Signum Biosciences, Inc. Acetyl mimic compounds for the inhibition of isoprenyl-S-cysteinyl methyltransferase
WO2010056778A1 (en) 2008-11-11 2010-05-20 Signum Biosciences, Inc. Isoprenyl compounds and methods thereof
EP2389357A4 (de) 2009-01-20 2013-01-23 Signum Biosciences Inc Entzündungshemmende komplexe

Also Published As

Publication number Publication date
ES2507515T3 (es) 2014-10-15
ES2333561T3 (es) 2010-02-23
US9265780B2 (en) 2016-02-23
JP2010270146A (ja) 2010-12-02
US20020156100A1 (en) 2002-10-24
US6583158B1 (en) 2003-06-24
US20050113415A1 (en) 2005-05-26
US20020035072A1 (en) 2002-03-21
EP1083899B1 (de) 2009-09-16
US6916829B2 (en) 2005-07-12
AU4186199A (en) 1999-12-20
US20150258129A1 (en) 2015-09-17
PT2143420E (pt) 2014-09-30
PT1083899E (pt) 2009-12-18
US20100137229A1 (en) 2010-06-03
CA2333965A1 (en) 1999-12-09
US6599919B2 (en) 2003-07-29
DK2143420T3 (da) 2014-10-06
US6589964B2 (en) 2003-07-08
DE69941429D1 (de) 2009-10-29
US20060100241A1 (en) 2006-05-11
US7141582B2 (en) 2006-11-28
US6274597B1 (en) 2001-08-14
US20030119874A1 (en) 2003-06-26
WO1999062517A1 (en) 1999-12-09
CY1112845T1 (el) 2016-02-10
DK1083899T3 (da) 2010-01-25
JP5528941B2 (ja) 2014-06-25
EP1083899A1 (de) 2001-03-21
CY1115583T1 (el) 2017-01-04
EP1083899A4 (de) 2004-11-03
US20160346262A1 (en) 2016-12-01
US20140206718A1 (en) 2014-07-24
US8436018B2 (en) 2013-05-07
US20040242539A1 (en) 2004-12-02
JP2003528799A (ja) 2003-09-30
US20020198225A1 (en) 2002-12-26
US8633221B2 (en) 2014-01-21
US7812033B2 (en) 2010-10-12
US20140051725A1 (en) 2014-02-20
US20110076765A1 (en) 2011-03-31
JP4602548B2 (ja) 2010-12-22
US7622485B2 (en) 2009-11-24
EP2143420B1 (de) 2014-06-25
EP2143420A1 (de) 2010-01-13
US20070021381A1 (en) 2007-01-25
US6774135B2 (en) 2004-08-10
CA2333965C (en) 2011-03-22
US7514453B2 (en) 2009-04-07
US8841322B2 (en) 2014-09-23

Similar Documents

Publication Publication Date Title
ATE442851T1 (de) 1-deoxy-galactonojirimycin-derivate zur vewendung bei der behandlung von lysosomalen speicherkrankheiten mittels steigerung der lysosomalen alpha galactosidase a
Murrell A radical proposal for the pathogenesis of scleroderma
ATE240744T1 (de) Verwendung von csf-1-inhibitoren
MX9803753A (es) Acidos alfa-iminohidroxamicos y -carboxilicos n-sustituidos ciclicos y heterociclicos.
ATE380559T1 (de) Konjugate enthaltend ein antitumorales mittel und deren verwendung
ATE189957T1 (de) Verwendung von nadph-oxidase-inhibitoren zur herstellung eines arzneimittels zur vorbeugung der arteriosklerose
TR199800818A2 (xx) �kame edilmi� diaminokarbonik asitler.
DE60032795D1 (de) Nordihydroguaiaretinsäurederivate zur behandlung von tumoren
DE60033684D1 (de) Phenylalaninderivate
DE69631157D1 (de) Pharmazeutische Zubereitung zur Verwendung in der Behandlung von Diabetes
ATE62414T1 (de) Mittel zur behandlung von herzerkrankungen.
ATE260931T1 (de) Verfahren zur identifizierung von verbindungen mit verminderter systemischer aktivität
ATE368455T1 (de) Verwendung von (+)-tramadol, o-demethyltramadol, o-desmethyl-n-mono-desmethyl-tramadol zur therapie der harninkontinenz
NO971341D0 (no) Promoter for reseptor tyrosinkinase TIE
KR950031085A (ko) 표피증식 질환 치료용 의약 조성물
ES2124861T3 (es) Derivados de aminoacidos y su utilizacion como inhibidores de la encefalinasa.
DE69916488D1 (de) Substituierte caprolactam-derivate, diese enthaltende pharmazeutische zubereitungen sowie ihre verwendung zur behandlung von tumoren
DK2359853T3 (da) Farmaceutiske sammensætninger og fremgangsmåder, der er anvendelige til at modulere antiogenese
DE69300037D1 (de) Verwendung von Bucillamin zur Herstellung eines Arzneimittels zur Behandlung von Cystinuria.
TR199901385T2 (xx) Piperidin t�revleri.
DE69813354D1 (de) Mittel zur Behandlung von Herzkrankheiten
ATE128358T1 (de) Therapeutische mittel für diabetisches gangrän.
ATE75938T1 (de) Enantiomere des propafenons enthaltende therapeutische mittel.
DE69825307D1 (de) Verwendung von protein h als zytostattischer wirkstoff
FI961029A (fi) Menetelmä maksasairauden ja vastaavien indikaatioiden hoitamiseksi vasodilatoivilla aineilla

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1083899

Country of ref document: EP

EEFA Change of the company name