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DK1083899T3 - Derivater af 1-dexoy-galactonojirimycin og anvendelse heraf i behandlingen af lysosomale oplagringssygdomme ved øgning af lysosomal alpha-galactosidase A - Google Patents

Derivater af 1-dexoy-galactonojirimycin og anvendelse heraf i behandlingen af lysosomale oplagringssygdomme ved øgning af lysosomal alpha-galactosidase A

Info

Publication number
DK1083899T3
DK1083899T3 DK99925611.8T DK99925611T DK1083899T3 DK 1083899 T3 DK1083899 T3 DK 1083899T3 DK 99925611 T DK99925611 T DK 99925611T DK 1083899 T3 DK1083899 T3 DK 1083899T3
Authority
DK
Denmark
Prior art keywords
lysosomal
galactonojirimycin
galactosidase
treatment
dexoy
Prior art date
Application number
DK99925611.8T
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 DK1083899T3 publication Critical patent/DK1083899T3/da

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)
  • Saccharide Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pyrrole Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
DK99925611.8T 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 DK1083899T3 (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
PCT/US1999/010479 WO1999062517A1 (en) 1998-06-01 1999-05-28 METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A

Publications (1)

Publication Number Publication Date
DK1083899T3 true DK1083899T3 (da) 2010-01-25

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 After (1)

Application Number Title Priority Date Filing Date
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

Country Status (12)

Country Link
US (16) US6274597B1 (da)
EP (2) EP1083899B1 (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 (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
EP1267914B2 (en) * 2000-02-04 2013-11-06 Children's Hospital Research Foundation Use of lysosomal acid lipase for treating atherosclerosis and related diseases
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
AU2003284886A1 (en) * 2002-10-21 2004-05-13 William E. Balch CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE
WO2006002283A1 (en) * 2004-06-21 2006-01-05 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
WO2004069190A2 (en) * 2003-01-31 2004-08-19 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
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
CA2507348C (en) * 2004-05-13 2013-07-16 The Hospital For Sick Children Real time methylumbelliferone-based assay
WO2005115429A1 (en) * 2004-05-24 2005-12-08 Ramot At Tel Aviv University Ltd. Methods and kits for diagnosing and/or assessing severity and treating gaucher disease
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
EP3441090A1 (en) * 2005-05-17 2019-02-13 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
MX2007015602A (es) * 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
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
EP2024745B1 (en) * 2006-05-16 2015-07-08 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
ES2364586T3 (es) * 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
MX2007006175A (es) * 2006-05-24 2009-02-16 Amicus Therapeutics Inc Sal de tartrato de isofagomina y metodos de uso.
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
WO2008088581A2 (en) * 2006-08-02 2008-07-24 United Therapeutics Corporation Liposome treatment of viral infections
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
JP2010523578A (ja) * 2007-03-30 2010-07-15 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るファブリー病の治療方法
MX2009011022A (es) * 2007-04-13 2010-01-13 Amicus Therapeutics Inc Uso de sustratos como chaperonas farmacologicas.
US8399525B2 (en) * 2007-04-13 2013-03-19 Amicus Therapeutics, Inc. Treatment of gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
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.
KR101650726B1 (ko) 2007-05-22 2016-08-24 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
WO2009049422A1 (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
SI2252313T1 (sl) * 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
US20090252785A1 (en) * 2008-03-26 2009-10-08 University Of Oxford Endoplasmic reticulum targeting liposomes
ES2963910T3 (es) 2008-06-26 2024-04-03 Zevra Denmark As Uso de Hsp70 como regulador de la actividad enzimática
WO2010048532A1 (en) * 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
US8703744B2 (en) * 2009-03-27 2014-04-22 The Chancellor, Masters And Scholars Of The University Of Oxford Cholesterol level lowering liposomes
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V 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
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
KR101880411B1 (ko) * 2009-10-19 2018-07-19 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
PL2490712T3 (pl) 2009-10-19 2015-12-31 Amicus Therapeutics Inc Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz
CN102647905B (zh) * 2009-10-19 2015-09-09 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
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
JP6170675B2 (ja) * 2010-01-28 2017-07-26 ラプトール ファーマシューティカルズ インコーポレイテッド 受容体関連タンパク質(rap)ペプチド−フコシダーゼ阻害薬複合体による肝障害処置のための方法
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
PT2646044T (pt) 2010-11-30 2019-11-12 Orphazyme As Métodos para aumentar a atividade intracelular de hsp70
EA031874B1 (ru) 2011-03-11 2019-03-29 Амикус Терапьютикс, Инк. Способ лечения болезни фабри
SI2753346T1 (sl) 2011-09-07 2020-09-30 Mount Sinai School Of Medicine Ceramidaza in diferenciacija celic
ES2639832T3 (es) 2011-11-29 2017-10-30 INSERM (Institut National de la Santé et de la Recherche Médicale) Métodos y composiciones farmacéuticas para el tratamiento de la enfermedad de Darier
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
LT2854910T (lt) 2012-06-01 2020-05-25 Icahn School Of Medicine At Mount Sinai Ceramido lygiai infekcijos gydymo ir prevencijos metu
ES2779006T3 (es) 2013-01-09 2020-08-13 Amicus Therapeutics Inc Composiciones de DNJ parenterales estables
HUE046113T2 (hu) 2013-03-14 2020-02-28 Icahn School Med Mount Sinai Terápiás sav ceramidáz készítmények és azok elõállítási és felhasználási eljárásai
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
MY186336A (en) 2014-09-30 2021-07-13 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates
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
EP3432882B1 (en) 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
IL301319A (en) 2016-03-30 2023-05-01 Amicus Therapeutics Inc Formulations that include recombinant acid alpha-glucosidase
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
RU2021117465A (ru) 2016-04-29 2021-07-22 Орфазим А/С Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений
TWI875676B (zh) 2016-07-19 2025-03-11 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
KR20240017111A (ko) 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
EP4374918B1 (en) 2017-05-30 2025-07-16 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
MY201196A (en) 2017-06-01 2024-02-09 Idorsia Pharmaceuticals Ltd Crystalline form of 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
PL4176877T3 (pl) 2017-08-28 2025-04-07 Amicus Therapeutics, Inc. Sposoby poprawy i/lub stabilizacji czynności serca u pacjentów z chorobą fabry'ego
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
US20190083242A1 (en) 2017-09-19 2019-03-21 Cardiovalve Ltd. Systems and methods for implanting a prosthetic valve within a native heart valve
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
LT3749308T (lt) 2018-02-06 2024-02-12 Amicus Therapeutics, Inc. Klasikine fabri liga sergančių pacientų gydymas migalastatu
WO2019157047A1 (en) 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
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
SI3840753T1 (sl) 2018-08-20 2025-01-31 Amicus Therapeutics, Inc. Migalastat za uporabo v postopkih zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA
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
MX2021015352A (es) 2019-06-11 2022-04-06 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal.
BR112022002202A2 (pt) 2019-08-07 2022-06-14 Amicus Therapeutics Inc Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla
US20220273615A1 (en) * 2019-10-04 2022-09-01 Academia Sinica Methods of treating pompe disease
IL293208A (en) * 2019-11-27 2022-07-01 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
HUE070116T2 (hu) 2020-03-06 2025-05-28 Amicus Therapeutics Inc Eljárások Fabry-kór kezelésére olyan betegekben, akiknek mutációja van a GLA génben
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
IL303026A (en) 2020-11-19 2023-07-01 Zevra Denmark As Processes for preparing arimoclomol citrate and intermediates thereof
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 (en) 2021-07-12 2024-05-22 Amicus Therapeutics, Inc. Methods of treating fabry disease in pediatric patients
US20240424137A1 (en) 2021-10-08 2024-12-26 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
WO1987003903A1 (en) 1985-12-23 1987-07-02 Fred Hutchinson Cancer Research Center Regulating retroviral replication, infection, and pathogenesis
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
EP0739205B1 (en) 1994-01-13 1999-11-24 G.D. Searle & Co. 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
EP0912179B1 (en) 1996-07-15 2006-04-12 MacroZyme DNM B.V. Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
CA2265464C (en) 1996-09-13 2007-06-26 Transkaryotic Therapies, Inc. 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
MY122499A (en) 1997-11-10 2006-04-29 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
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
EE200100302A (et) 1998-12-02 2002-08-15 Pfizer Products Inc. Meetodid ja kompositsioonid p53 perekonna valgu konformatsioonilise stabiilsuse taastamiseks
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
EP1196190B1 (en) 1999-07-26 2003-03-19 G.D. SEARLE &amp; CO. Use of long-chain n-alkyl derivatives of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of a medicament for the treatment of glycolipid storage diseases
JP2003506406A (ja) 1999-08-10 2003-02-18 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 長鎖n−アルキル化合物およびそのオキサ誘導体
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
WO2001052830A2 (en) 2000-01-20 2001-07-26 Washington University METHODS TO TREAT α-1-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
US20020115619A1 (en) 2000-10-04 2002-08-22 Rubenstein Ronald C. 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
PT2362866E (pt) 2008-11-11 2015-10-26 Signum Biosciences Inc Compostos de isoprenila e métodos dos mesmos
JP2012515721A (ja) 2009-01-20 2012-07-12 シグナム バイオサイエンシーズ, インコーポレイテッド 抗炎症性複合体

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