DK2143420T3 - Formulering, der omfatter mutant(er) af lysosomal alfa-galactosidase A, til behandling af Fabrys sygdom - Google Patents

Formulering, der omfatter mutant(er) af lysosomal alfa-galactosidase A, til behandling af Fabrys sygdom

Info

Publication number
DK2143420T3
DK2143420T3 DK09011761.5T DK09011761T DK2143420T3 DK 2143420 T3 DK2143420 T3 DK 2143420T3 DK 09011761 T DK09011761 T DK 09011761T DK 2143420 T3 DK2143420 T3 DK 2143420T3
Authority
DK
Denmark
Prior art keywords
galactosidase
treatment
fabry
disease
mutant
Prior art date
Application number
DK09011761.5T
Other languages
English (en)
Inventor
Satoshi Ishi
Jian-Qiang Fan
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 DK2143420T3 publication Critical patent/DK2143420T3/da

Links

Classifications

    • 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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic 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
    • C07D451/02—Heterocyclic 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
    • C07D451/04—Heterocyclic 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
    • C07D451/06—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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)
DK09011761.5T 1998-06-01 1999-05-28 Formulering, der omfatter mutant(er) af lysosomal alfa-galactosidase A, til behandling af Fabrys sygdom DK2143420T3 (da)

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
EP99925611A EP1083899B1 (en) 1998-06-01 1999-05-28 Derivatives of 1-deoxy-galactonojirimycin and their use in the treatment of lysosomal storage diseases by enhancing lysosomal alpha-galactosidase a

Publications (1)

Publication Number Publication Date
DK2143420T3 true DK2143420T3 (da) 2014-10-06

Family

ID=22207362

Family Applications (2)

Application Number Title Priority Date Filing Date
DK99925611.8T DK1083899T3 (da) 1998-06-01 1999-05-28 Derivater af 1-dexoy-galactonojirimycin og anvendelse heraf i behandlingen af lysosomale oplagringssygdomme ved øgning af lysosomal alpha-galactosidase A
DK09011761.5T DK2143420T3 (da) 1998-06-01 1999-05-28 Formulering, der omfatter mutant(er) af lysosomal alfa-galactosidase A, til behandling af Fabrys sygdom

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK99925611.8T DK1083899T3 (da) 1998-06-01 1999-05-28 Derivater af 1-dexoy-galactonojirimycin og anvendelse heraf i behandlingen af lysosomale oplagringssygdomme ved øgning af lysosomal alpha-galactosidase A

Country Status (12)

Country Link
US (16) US6274597B1 (da)
EP (2) EP2143420B1 (da)
JP (2) JP4602548B2 (da)
AT (1) ATE442851T1 (da)
AU (1) AU4186199A (da)
CA (1) CA2333965C (da)
CY (2) CY1112845T1 (da)
DE (1) DE69941429D1 (da)
DK (2) DK1083899T3 (da)
ES (2) ES2507515T3 (da)
PT (2) PT2143420E (da)
WO (1) WO1999062517A1 (da)

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
BR0108077A (pt) * 2000-02-04 2002-10-22 Children S Hospital Res Founda Processo para reduzir placas ateroscleróticas em um mamìfero, método para tratamento da aterosclerose em um mamìfero, composição, métodos para prover proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo ou mistura deles, para células de um mamìfero tendo deficiência na proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo , para prover lipase de ácido lisossomal biologicamente ativa para células de um mamìfero tendo deficiência de lipase de ácido lisossomal biologicamente ativa, para prover lipase de ácido lisossoal biologicamente ativa para células de um mamìfero com aterosclerose, para tratamento da doença de wolman em um mamìfero, para tratamento de doença de armazenagem de colesteril éster em um mamìfero, e, para tratamento da aterosclerose em um mamìfero
ATE496020T1 (de) * 2001-09-07 2011-02-15 Seikagaku Kogyo Co Ltd Carbazuckeraminderivate und mittel gegen störungen des glycolipidmetabolismus, die diese als wirkstoff 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
WO2004037373A2 (en) * 2002-10-21 2004-05-06 The Scripps Research Institute CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE
WO2006002283A1 (en) * 2004-06-21 2006-01-05 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
CA2814774C (en) 2003-01-31 2016-03-22 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
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
BRPI0416217A8 (pt) * 2003-11-12 2018-05-08 Amicus Therapeutics Inc composto derivado de hidroxipiperidina, métodos para a síntese de um composto derivado de hidroxipiperidina para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase numa célula de mamífero e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir beta-glicosidade, e para tratar doença de gaucher e derivado hidroxipiperidina com ligante flexível curto
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
WO2006007560A2 (en) * 2004-07-01 2006-01-19 University Of Pennsylvania 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 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US20070021433A1 (en) 2005-06-03 2007-01-25 Jian-Qiang Fan Pharmacological chaperones for treating obesity
KR20080033242A (ko) * 2005-06-08 2008-04-16 아미쿠스 세라퓨틱스, 인코포레이티드 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는ｃｎｓ 질환의 치료 방법
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
WO2007030375A2 (en) * 2005-09-08 2007-03-15 Children's 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
CA2652553C (en) * 2006-05-16 2020-08-18 Amicus Therapeutics, Inc. Use of 1-deoxygalactonojirimycin or a salt thereof in the treatment of fabry disease
ATE504570T1 (de) * 2006-05-24 2011-04-15 Amicus Therapeutics Inc Tartratsalz aus isofagomin und verwendungsverfahren dafür
ES2364586T3 (es) * 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
CA2656643C (en) * 2006-06-23 2015-08-25 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase
JP2009545621A (ja) * 2006-08-02 2009-12-24 ユナイテッド セラピューティクス コーポレーション ウィルス感染症のリポソーム処置
EP2121005A1 (en) * 2006-12-21 2009-11-25 Academisch Ziekenhuis bij de Universiteit van Amsterdam Treatment of fabry disease
WO2008112525A2 (en) * 2007-03-09 2008-09-18 Link Medicine Corporation Treatment of lysosomal storage diseases
US20100113517A1 (en) * 2007-03-30 2010-05-06 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
WO2008128089A1 (en) * 2007-04-13 2008-10-23 Amicus Therapeutics, Inc. Use of substrates as pharmacological chaperones
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
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.
AU2008254594B2 (en) 2007-05-22 2013-06-27 Amicus Therapeutics, Inc. New method for preparing isofagomine and its derivatives
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
US20100317690A1 (en) * 2007-11-21 2010-12-16 Summit Corporation Plc Treatment of protein folding disorders
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CN102046151A (zh) * 2008-03-26 2011-05-04 牛津大学 靶向内质网的脂质体
WO2009155936A1 (en) 2008-06-26 2009-12-30 Orphazyme Aps Use of hsp70 as a regulator of enzymatic activity
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
JP2012521981A (ja) * 2009-03-27 2012-09-20 ザ チャンセラー，マスターズ アンド スカラーズ オブ ザ ユニバーシティ オブ オックスフォード コレステロールレベル低下リポソーム
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
AU2010254092B2 (en) * 2009-05-26 2015-11-12 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US20110112041A1 (en) * 2009-10-01 2011-05-12 Baylor Research Institute Treatment of Male-Pattern Baldness by Local Induction of the Metabolic Defect of Fabry Disease
JP6061677B2 (ja) 2009-10-19 2017-01-18 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てガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
CN105193802A (zh) 2009-10-19 2015-12-30 阿米库斯治疗学公司 用于预防和/或治疗溶酶体贮积失调的新颖组合物
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
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
AU2011210756B2 (en) * 2010-01-28 2016-11-03 Horizon Orphan Llc Method For Treating Liver Disorders With Receptor Associated Protein (RAP) Peptide-Fucosidase Inhibitor Conjugates
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
JP2013544263A (ja) 2010-11-30 2013-12-12 オルファザイム エーピーエス Ｈｓｐ７０の細胞内活性を上昇させる方法
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
SI2753346T1 (sl) 2011-09-07 2020-09-30 Mount Sinai School Of Medicine Ceramidaza in diferenciacija celic
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
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2897825T3 (es) 2012-06-01 2022-03-02 Icahn School Med Mount Sinai Niveles de ceramida en el tratamiento y prevención de infecciones
ES2779006T3 (es) 2013-01-09 2020-08-13 Amicus Therapeutics Inc Composiciones de DNJ parenterales estables
EP2968479B1 (en) 2013-03-14 2019-07-17 Icahn School of Medicine at Mount Sinai Therapeutic acid ceramidase compositions and methods of making and using them
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
CN107075468B (zh) 2014-09-30 2021-12-24 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
EP3253333B1 (en) 2015-02-05 2024-04-03 Cardiovalve 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
KR102004114B1 (ko) * 2016-03-22 2019-07-26 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
IL295551A (en) 2016-03-30 2022-10-01 Amicus Therapeutics Inc A method for selecting recombinant proteins with a high amount of mannose-6-phosphate
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
HUE052158T2 (hu) 2016-04-29 2021-04-28 Orphazyme As Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez
US20190183869A1 (en) 2016-07-19 2019-06-20 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve 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.
CN111278438A (zh) 2017-05-30 2020-06-12 阿米库斯治疗学公司 治疗具有肾损害的法布里患者的方法
CA3224546A1 (en) 2017-05-30 2018-12-06 Jeff Castelli Use of migalastat in the treatment of fabry patients having renal impairment
JP6937850B2 (ja) 2017-06-01 2021-09-22 イドーシア ファーマシューティカルズ リミテッドＩｄｏｒｓｉａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｌｔｄ Ｎ−ブチルデオキシガラクトノジリマイシンの結晶形
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
US11819405B2 (en) 2017-09-19 2023-11-21 Cardiovalve Ltd. Prosthetic valve with inflatable cuff configured for radial extension
RS63742B1 (sr) 2017-08-28 2022-12-30 Amicus Therapeutics Inc Metode unapređenja i/ili stabilizacije srčanih funkcija kod pacijenata sa fabrijevom bolešću
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
PL3749308T3 (pl) 2018-02-06 2024-03-25 Amicus Therapeutics, Inc. Leczenie pacjentów z klasyczną chorobą fabry’ego migalastatem
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP4285990A3 (en) 2018-08-20 2024-02-21 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
KR20220019796A (ko) 2019-06-11 2022-02-17 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
EP4037682A4 (en) * 2019-10-04 2023-11-01 Academia Sinica METHODS OF TREATMENT OF PUMP DISEASE
IL293208A (en) * 2019-11-27 2022-07-01 Turning Point Therapeutics Inc Combination therapy involving diaryl macrocyclic compounds
WO2021163183A1 (en) * 2020-02-10 2021-08-19 Amicus Therapeutics, Inc. Methods of treating fabry disease
AU2021230371A1 (en) 2020-03-06 2022-09-29 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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
CA3225511A1 (en) 2021-07-12 2023-01-19 Franklin Johnson Methods of treating fabry disease in pediatric patients
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
EP4385509A1 (en) 2022-12-13 2024-06-19 Amicus Therapeutics, Inc. Migalastat formulations having improved pharmacokinetics

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
EP0536402A4 (en) 1990-06-29 1993-05-12 Nippon Shinyaku Company, Limited 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
US5798366A (en) * 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US6291657B1 (en) * 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives
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
CA2181033C (en) 1994-01-13 2007-09-04 Timothy M. Block Use of n-alkyl derivatives of 1,5-dideoxy-1,5-imino-d-glucitol for the treatment of hepatitis b virus infections
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
JP4564108B2 (ja) 1996-07-15 2010-10-20 ジェンザイム コーポレーション デオキシノジリマイシン誘導体及び該誘導体のグルコシルセラミダーゼ阻害剤としての用途
WO1998011206A2 (en) 1996-09-13 1998-03-19 Transkaryotic Therapies, Inc. THERAPY FOR α-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
DK1030839T3 (da) 1997-11-10 2004-05-03 Searle & Co Anvendelse af alkylerede iminosukkere til behandling af multidrugresistens
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
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
AU1290700A (en) 1998-12-02 2000-06-19 Pfizer Products Inc. Methods and compositions for restoring conformational stability of a protein of the p53 family
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
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
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
WO2001021652A1 (en) 1999-09-24 2001-03-29 The Johns Hopkins University School Of Medicine Methods for identifying an agent that corrects defective protein folding
EP1250129A2 (en) 2000-01-20 2002-10-23 Washington University METHODS TO TREAT $g(a)-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
AU2002211379A1 (en) 2000-10-04 2002-04-15 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
CN101945652A (zh) 2008-02-14 2011-01-12 西格纳姆生物科学公司 抑制异戊二烯基-s-半胱氨酰基甲基转移酶的乙酰基模拟化合物
ES2549158T3 (es) 2008-11-11 2015-10-23 Signum Biosciences, Inc. Compuestos de isoprenilo y métodos de los mismos
EP2389357A4 (en) 2009-01-20 2013-01-23 Signum Biosciences Inc INFLAMMATORY COMPLEXES

Also Published As

Publication number Publication date
CY1112845T1 (el) 2016-02-10
US6583158B1 (en) 2003-06-24
US7812033B2 (en) 2010-10-12
US20030119874A1 (en) 2003-06-26
US20020035072A1 (en) 2002-03-21
US8633221B2 (en) 2014-01-21
US6589964B2 (en) 2003-07-08
US6774135B2 (en) 2004-08-10
ES2333561T3 (es) 2010-02-23
US6916829B2 (en) 2005-07-12
PT2143420E (pt) 2014-09-30
US8841322B2 (en) 2014-09-23
US20100137229A1 (en) 2010-06-03
US7622485B2 (en) 2009-11-24
US20140206718A1 (en) 2014-07-24
US7514453B2 (en) 2009-04-07
US20110076765A1 (en) 2011-03-31
EP2143420A1 (en) 2010-01-13
EP1083899B1 (en) 2009-09-16
AU4186199A (en) 1999-12-20
US6274597B1 (en) 2001-08-14
EP1083899A4 (en) 2004-11-03
DK1083899T3 (da) 2010-01-25
EP1083899A1 (en) 2001-03-21
US6599919B2 (en) 2003-07-29
CA2333965A1 (en) 1999-12-09
PT1083899E (pt) 2009-12-18
CA2333965C (en) 2011-03-22
US20070021381A1 (en) 2007-01-25
WO1999062517A1 (en) 1999-12-09
JP2003528799A (ja) 2003-09-30
JP4602548B2 (ja) 2010-12-22
JP5528941B2 (ja) 2014-06-25
ES2507515T3 (es) 2014-10-15
US7141582B2 (en) 2006-11-28
US20160346262A1 (en) 2016-12-01
CY1115583T1 (el) 2017-01-04
US20060100241A1 (en) 2006-05-11
EP2143420B1 (en) 2014-06-25
US20150258129A1 (en) 2015-09-17
US20020198225A1 (en) 2002-12-26
DE69941429D1 (de) 2009-10-29
US9265780B2 (en) 2016-02-23
US20050113415A1 (en) 2005-05-26
JP2010270146A (ja) 2010-12-02
US20140051725A1 (en) 2014-02-20
US20040242539A1 (en) 2004-12-02
ATE442851T1 (de) 2009-10-15
US20020156100A1 (en) 2002-10-24
US8436018B2 (en) 2013-05-07

Similar Documents

Publication Publication Date Title
DK2143420T3 (da) Formulering, der omfatter mutant(er) af lysosomal alfa-galactosidase A, til behandling af Fabrys sygdom
ZA907338B (en) Thienylcarboxylates of amino alcohols
PL336580A1 (en) Fine-molecular compounds useful in treating inflammatory diseases
NO308854B1 (no) Terapeutiske forbindelser, farmasøytisk akseptable salter og formuleringer inneholdende forbindelsene, samt anvendelser derav
DK233586A (da) 5-substituerede 1,3-thiazolidon-2,4-dion forbindelser og farmaceutiske praeparater indeholdende disse
CY2473B1 (en) New phenanthridines.
IT1307047B1 (it) Apparecchiatura per il trattamento umidificato dell&#39;apnea da sonno.
DK0774005T3 (da) Overfladeudtrykkelse af enzymer i genorienteret prodrug-terapi
ES2184077T3 (es) Nuevas fenantridinas sustituidas en la posicion 6.
IT1141249B (it) Procedimento per la preparazione di copolimeri di polisaccaridi supprotanti attivita&#39; enzimatica,e copolimeri cosi&#39; ottenuti
TR200002248T2 (tr) Yeni dihidroksiheksanoik asit türevleri
EE9900477A (et) Ühendid ja kompositsioonid seriinproteaasse, eriti trüptaasse aktiivsusega seotud haiguste raviks
DE59509395D1 (de) Verwendung von Lactamverbindungen als pharmazeutische Wirkstoffe
DK393188A (da) Farmaceutisk praeparat til intranasal administrering
WO1997009317A3 (en) Optically active phenyl pyrimidine derivative as analgesic agent
PT783495E (pt) Derivados de quinoxalina uteis em terapia
NO874241D0 (no) Terapeutisk aktive salter.
ATE106732T1 (de) Arzneimittelzubereitung, enthaltend stigmasta-4- en-3-on sowie deren verwendung.
ES2141351T3 (es) Nuevo principio activo peptidico y su obtencion.
FR2517679B1 (da)
YU58900A (sh) Lečenje paničnih napada
FI922864A0 (fi) Terapeutiskt aktiva fluorsubsti- tuerade bensimidazoler, foerfaranden foer deras framstaellning samt deras anvaendning.
FR2715849B1 (fr) Forme d&#39;administration solide à délitescence rapide d&#39;un principe actif.
IT1213536B (it) 2-bromo,1&#39;,2&#39;,-dicloro-perfluoro dietil-etere e procedimento per lasua preparazione.
NO880639D0 (no) Fremgangsmaate for fremstilling av terapeutisk aktive oksimetere av 2,6-dioksabicyklo (3, 3, 0)oktanoner.