JP4602548B2 - リソソームα−ガラクトシダーゼAの増強方法 - Google Patents

リソソームα−ガラクトシダーゼAの増強方法 Download PDF

Info

Publication number
JP4602548B2
JP4602548B2 JP2000551773A JP2000551773A JP4602548B2 JP 4602548 B2 JP4602548 B2 JP 4602548B2 JP 2000551773 A JP2000551773 A JP 2000551773A JP 2000551773 A JP2000551773 A JP 2000551773A JP 4602548 B2 JP4602548 B2 JP 4602548B2
Authority
JP
Japan
Prior art keywords
dgj
gala
enzyme
cells
activity
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
JP2000551773A
Other languages
English (en)
Japanese (ja)
Other versions
JP2003528799A5 (enExample
JP2003528799A (ja
Inventor
ファン、ジアン・キアン
達 石井
Original Assignee
マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー
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 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー filed Critical マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー
Publication of JP2003528799A publication Critical patent/JP2003528799A/ja
Publication of JP2003528799A5 publication Critical patent/JP2003528799A5/ja
Application granted granted Critical
Publication of JP4602548B2 publication Critical patent/JP4602548B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Images

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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Hydrogenated Pyridines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pyrrole Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
JP2000551773A 1998-06-01 1999-05-28 リソソームα−ガラクトシダーゼAの増強方法 Expired - Lifetime JP4602548B2 (ja)

Applications Claiming Priority (3)

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
US09/087,804 1998-06-01
PCT/US1999/010479 WO1999062517A1 (en) 1998-06-01 1999-05-28 METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2010173733A Division JP5528941B2 (ja) 1998-06-01 2010-08-02 リソソームα−ガラクトシダーゼAの増強方法

Publications (3)

Publication Number Publication Date
JP2003528799A JP2003528799A (ja) 2003-09-30
JP2003528799A5 JP2003528799A5 (enExample) 2006-07-13
JP4602548B2 true JP4602548B2 (ja) 2010-12-22

Family

ID=22207362

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2000551773A Expired - Lifetime JP4602548B2 (ja) 1998-06-01 1999-05-28 リソソームα−ガラクトシダーゼAの増強方法
JP2010173733A Expired - Lifetime JP5528941B2 (ja) 1998-06-01 2010-08-02 リソソームα−ガラクトシダーゼAの増強方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2010173733A Expired - Lifetime JP5528941B2 (ja) 1998-06-01 2010-08-02 リソソームα−ガラクトシダーゼAの増強方法

Country Status (12)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010270146A (ja) * 1998-06-01 2010-12-02 Mount Sinai School Of Medicine Of New York Univ リソソームα−ガラクトシダーゼAの増強方法

Families Citing this family (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2003022797A1 (en) * 2001-09-07 2003-03-20 Seikagaku Corporation Carba-sugar amine derivatives and treatments for disorder of glycolipid metabolism containing the same as the active ingredient
US7842470B2 (en) * 2001-10-09 2010-11-30 Oregon Health & Science University Method for pharmacoperones correction of GnRHR mutant protein misfolding
AU2003281195A1 (en) * 2002-07-05 2004-01-23 Roderick Frederick Gerardus Joseph King 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
PT2444102E (pt) * 2003-01-31 2015-09-17 Sinai School Medicine Terapia combinada para o tratamento de distúrbios de deficiência proteica
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP1633356A1 (en) * 2003-05-22 2006-03-15 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
US20050137223A1 (en) * 2003-11-12 2005-06-23 Amicus Therapeutics, Inc. Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease
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
CA2568150A1 (en) * 2004-06-21 2006-01-05 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
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
EP2932982B1 (en) 2005-05-17 2018-10-03 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
AU2006254796B2 (en) * 2005-06-08 2012-08-16 Amicus Therapeutics, Inc. Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes
FR2887549B1 (fr) * 2005-06-23 2009-07-10 Centre Nat Rech Scient Nouveaux composes de la famille des iminosucres, leurs utilisations, notamment pour le traitement de maladies lysosomales, ainsi que leur procede de preparation
WO2007030375A2 (en) * 2005-09-08 2007-03-15 Children's Hospital Medical Center Lysosomal acid lipase therapy for nafld and related diseases
AU2015230773B2 (en) * 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
FI3457135T3 (fi) * 2006-05-16 2025-03-08 Amicus Therapeutics Inc Fabryn taudin hoitovaihtoehtoja
WO2007140212A2 (en) * 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
ES2364586T3 (es) * 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
KR101402554B1 (ko) * 2006-06-23 2014-06-19 아미쿠스 세라퓨틱스, 인코포레이티드 β-글루코세레브로시다제의 활성 증진에 의한 신경계 장애 치료 방법
EP2356990A3 (en) * 2006-08-02 2011-10-19 University of Oxford Liposome treatment of viral infections
EP2121005A1 (en) * 2006-12-21 2009-11-25 Academisch Ziekenhuis bij de Universiteit van Amsterdam Treatment of fabry disease
EP2155197A4 (en) * 2007-03-09 2011-10-12 Link Medicine Corp TREATMENT OF LYSOSOMAL STORAGE DISEASES
WO2008121826A2 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using 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
WO2008128089A1 (en) * 2007-04-13 2008-10-23 Amicus Therapeutics, Inc. Use of substrates as pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
KR101597499B1 (ko) 2007-05-22 2016-02-24 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
WO2009049421A1 (en) * 2007-10-18 2009-04-23 The Hospital For Sick Children Compositions and methods for enhancing enzyme activity in gaucher, gm1-gangliosidosis/morquio b disease, and parkinson's disease
EP2252288A1 (en) * 2007-11-21 2010-11-24 Summit Corporation Plc Treatment of protein folding disorders
HUE026543T2 (hu) 2008-02-12 2016-06-28 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
WO2009118658A2 (en) * 2008-03-26 2009-10-01 University Of Oxford Endoplasmic reticulum targeting liposomes
PL2659904T3 (pl) 2008-06-26 2016-01-29 Orphazyme Aps Zastosowanie Hsp70 jako regulatora aktywności enzymatycznej
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
EP2435459B1 (en) * 2009-05-26 2016-05-04 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
TW201117810A (en) * 2009-10-01 2011-06-01 Baylor Res Inst Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
RU2015147509A (ru) * 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
WO2011049787A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
EP2490533B1 (en) 2009-10-19 2015-09-16 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
CA2788175A1 (en) * 2010-01-28 2011-08-04 Raptor Discoveries Inc. Method for treating liver disorders with receptor associated protein (rap) peptide-fucosidase inhibitor conjugates
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
DK2646044T3 (da) 2010-11-30 2019-11-25 Orphazyme As Metoder til at øge den intracellulære aktivitet af Hsp70
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
WO2013036875A1 (en) 2011-09-07 2013-03-14 Mount Sinai School Of Medicine Ceramidase and cell differentiation
EP2785335B8 (en) 2011-11-29 2017-08-02 Institut National de la Sante et de la Recherche Medicale (INSERM) Methods and pharmaceutical compositions for the treatment of darier disease
KR20140135222A (ko) 2012-03-07 2014-11-25 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
WO2014110270A1 (en) 2013-01-09 2014-07-17 Amicus Therapeutics, Inc. Stable parenteral dnj compositions
WO2014160390A1 (en) 2013-03-14 2014-10-02 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
KR20250069686A (ko) 2014-09-15 2025-05-19 제브라 덴마크 에이/에스 아리모클로몰 제제
SI4273241T1 (sl) 2014-09-30 2025-03-31 Amicus Therapeutics, Inc. Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati
WO2016125160A1 (en) 2015-02-05 2016-08-11 Mitraltech Ltd. Prosthetic valve with axially-sliding frames
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
WO2017165164A1 (en) * 2016-03-22 2017-09-28 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
NZ786723A (en) 2016-03-30 2025-07-25 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
LT3448382T (lt) 2016-04-29 2021-04-12 Orphazyme A/S Arimoklomolis, skirtas su gliukocerebrozidaze susijusiems sutrikimams gydyti
CA3031249A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
DK3624831T5 (da) 2017-05-15 2024-09-02 Amicus Therapeutics Inc Rekombinant human sur alfa-glucosidase
TWI795408B (zh) 2017-05-30 2023-03-11 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
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
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
LT4176877T (lt) 2017-08-28 2025-02-10 Amicus Therapeutics, Inc. Pacientų, sergančių fabri liga, širdies funkcijos gerinimo ir (arba) stabilizavimo būdai
US20200206323A1 (en) * 2017-09-08 2020-07-02 The Nemours Foundation An agent, a device and a blood-circulation system for treating lysosomal storage diseases, and a method for treating lysosomal storage diseases
US11304806B2 (en) 2017-09-19 2022-04-19 Cardiovalve Ltd. Prosthetic valve with atrial tissue anchors having variable flexibility and ventricular tissue anchors having constant flexibility
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
EP4316589A3 (en) * 2018-02-06 2024-04-24 Amicus Therapeutics, Inc. Treatment of patients with classic fabry disease
CA3090496C (en) 2018-02-06 2024-03-26 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
PL3840753T3 (pl) 2018-08-20 2025-01-20 Amicus Therapeutics, Inc. Migalastat do zastosowania w sposobach leczenia choroby fabry'ego u pacjentów mających mutację w genie 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
AR120055A1 (es) 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
EP4037682A4 (en) * 2019-10-04 2023-11-01 Academia Sinica METHOD FOR TREATING POMPE'S DISEASE
KR20220134522A (ko) * 2019-11-27 2022-10-05 터닝 포인트 테라퓨틱스, 인크. 디아릴 매크로시클릭 화합물을 수반하는 병용 요법
CA3167476A1 (en) * 2020-02-10 2021-08-19 Nina SKUBAN Methods of treating fabry disease
RS66530B1 (sr) 2020-03-06 2025-03-31 Amicus Therapeutics Inc Metode lečenja fabrijeve bolesti kod pacijenata koji imaju mutaciju u gla genu
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
US20230416224A1 (en) 2020-11-19 2023-12-28 Zevra Denmark A/S 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
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 (en) 2021-10-08 2024-08-14 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
DE3650377T2 (de) 1985-12-23 1996-02-22 Hutchinson Fred Cancer Res Regulierung der retroviralen replikation, infektion und pathogenese.
DE3737523A1 (de) 1987-11-05 1989-05-18 Bayer Ag Verwendung von substituierten hydroxypiperidinen als antivirale mittel
US5192772A (en) 1987-12-09 1993-03-09 Nippon Shinyaku Co. Ltd. Therapeutic agents
US5250545A (en) 1988-02-12 1993-10-05 Meiji Seika Kaisha, Ltd. Cancer cell metastasis inhibitor methods
FR2640621B1 (fr) 1988-12-19 1992-10-30 Centre Nat Rech Scient N-aryl-azetidinones, leur procede de preparation et leur utilisation comme inhibiteurs des elastases
US5043273A (en) 1989-08-17 1991-08-27 Monsanto Company Phosphorylated glycosidase inhibitor prodrugs
US5030638A (en) 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US5504078A (en) 1990-06-08 1996-04-02 Merrell Dow Pharmaceuticals Inc. α-glucosidase inhibitors
WO1992000277A1 (en) 1990-06-29 1992-01-09 Nippon Shinyaku Co., Ltd. Piperidine derivative
US5276120A (en) 1991-05-30 1994-01-04 The Scripps Research Institute Process for forming omega-deuxy-azasugars
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
US5399567A (en) * 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
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
DE69421828T2 (de) 1994-01-13 2000-04-20 G.D. Searle & Co. Verwendung von 1,5-dideoxy-1,5-imino-d-glucitol n-alkylderivaten zur behandlung von hepatitis b infektionen
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
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
DK2374876T3 (en) 1996-09-13 2016-05-30 Shire Human Genetic Therapies A process for the purification of alpha-galactosidase A
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
AU753336B2 (en) 1997-11-10 2002-10-17 G.D. Searle & Co. Use of alkylated iminosugars to treat multidrug resistance
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
WO1999040435A1 (en) 1998-02-09 1999-08-12 Netzer William J Protein folding inhibitors
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
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
KR20010086073A (ko) 1998-12-02 2001-09-07 데이비드 존 우드 p53계 단백질의 배좌 안정성을 회복하기 위한 방법 및조성물
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
JP2002542202A (ja) 1999-04-19 2002-12-10 シェリング・コーポレーション 抗酸化剤を伴ったリバビリンを含有するhcv組み合わせ治療薬
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
AU1840101A (en) 1999-08-10 2001-03-05 Chancellor, Masters And Scholars Of The University Of Oxford, The Long chain n-alkyl compounds and oxo-derivatives thereof
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
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation 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
WO2009102997A2 (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
CA2787339A1 (en) 2009-01-20 2011-08-12 Signum Biosciences, Inc. Anti-inflammatory complexes

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010270146A (ja) * 1998-06-01 2010-12-02 Mount Sinai School Of Medicine Of New York Univ リソソームα−ガラクトシダーゼAの増強方法

Also Published As

Publication number Publication date
WO1999062517A1 (en) 1999-12-09
US9265780B2 (en) 2016-02-23
US6599919B2 (en) 2003-07-29
US6274597B1 (en) 2001-08-14
JP2010270146A (ja) 2010-12-02
US6774135B2 (en) 2004-08-10
DE69941429D1 (de) 2009-10-29
ES2333561T3 (es) 2010-02-23
AU4186199A (en) 1999-12-20
US20140206718A1 (en) 2014-07-24
EP1083899A4 (en) 2004-11-03
US20110076765A1 (en) 2011-03-31
CY1115583T1 (el) 2017-01-04
CA2333965C (en) 2011-03-22
EP1083899B1 (en) 2009-09-16
US7141582B2 (en) 2006-11-28
US7622485B2 (en) 2009-11-24
US20040242539A1 (en) 2004-12-02
US20060100241A1 (en) 2006-05-11
US20160346262A1 (en) 2016-12-01
EP1083899A1 (en) 2001-03-21
DK2143420T3 (da) 2014-10-06
ES2507515T3 (es) 2014-10-15
JP2003528799A (ja) 2003-09-30
US20070021381A1 (en) 2007-01-25
US20050113415A1 (en) 2005-05-26
US20140051725A1 (en) 2014-02-20
US20020156100A1 (en) 2002-10-24
PT1083899E (pt) 2009-12-18
EP2143420A1 (en) 2010-01-13
PT2143420E (pt) 2014-09-30
US7514453B2 (en) 2009-04-07
US20030119874A1 (en) 2003-06-26
US20100137229A1 (en) 2010-06-03
CY1112845T1 (el) 2016-02-10
US8841322B2 (en) 2014-09-23
US7812033B2 (en) 2010-10-12
US8436018B2 (en) 2013-05-07
US8633221B2 (en) 2014-01-21
EP2143420B1 (en) 2014-06-25
DK1083899T3 (da) 2010-01-25
US20020198225A1 (en) 2002-12-26
US6583158B1 (en) 2003-06-24
ATE442851T1 (de) 2009-10-15
CA2333965A1 (en) 1999-12-09
US6589964B2 (en) 2003-07-08
US20020035072A1 (en) 2002-03-21
JP5528941B2 (ja) 2014-06-25
US6916829B2 (en) 2005-07-12
US20150258129A1 (en) 2015-09-17

Similar Documents

Publication Publication Date Title
JP4602548B2 (ja) リソソームα−ガラクトシダーゼAの増強方法
Ritter et al. Expression and inducibility of the human bilirubin UDP‐glucuronosyltransferase UGT1A1 in liver and cultured primary hepatocytes: Evidence for both genetic and environmental influences
US9925271B2 (en) Targeted pharmacological chaperones
US20100152125A1 (en) Compositions And Methods For The Diagnosis, Treatment, And Prevention Of Amyotrophic Lateral Sclerosis And Related Neurological Diseases
US20250177361A1 (en) Compositions and methods for treating neurodegenerative, myodegenerative, and lysosomal storage disorders
Lei et al. Enzyme enhancement activity of N-octyl-β-valienamine on β-glucosidase mutants associated with Gaucher disease
US6149909A (en) Synthetic α-L-iduronidase and genetic sequences encoding same
US4599331A (en) Etianic acids as antiangiogenics
Amendt et al. Short-chain acyl-coenzyme A dehydrogenase activity, antigen, and biosynthesis are absent in the BALB/cByJ mouse
US10519157B2 (en) Deuterated compounds for treating Fabry, Gaucher, Parkinson&#39;s and related diseases and conditions, and compositions and methods thereof
WO2018149864A1 (en) Small molecule therapeutics for treating metachromatic leukodystrophy
US7341990B2 (en) Methods and uses of leukotriene B4 hydroxylases to treat diseases
Qi et al. Ex vivo localization of the mouse saposin C activation region for acid β-glucosidase
WO2021188296A1 (en) Methods of treating xanthine oxidase-related diseases with niflumic acid and derivatives thereof
WO2014172776A1 (en) Compounds for the treatment of mucopolysaccharidoses
WO1997025043A1 (en) A method of treating hypercholesterolemia and related disorders
HK1069326A1 (en) Use of thiazole derivatives for preparing a medicine for protecting mitochondria
HK1069326B (en) Use of thiazole derivatives for preparing a medicine for protecting mitochondria
WO2014102417A1 (es) UTILIZACIÓN DE DERIVADOS BICÍCLICOS DE 1-DESOXIGALACTONOJIRIMICINA EN LA PREPARACIÓN DE UN MEDICAMENTO PARA EL TRATAMIENTO DE ENFERMEDADES RELACIONADAS CON ß-ENZIMAS GALACTOSIDASAS LISOSÓMICAS MUTANTES HUMANAS
NZ280680A (en) Use of a P450 1A inhibitor to inhibit the enzymatic metabolism of a nitrogen substituted acridine derivative

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20060526

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20060526

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20100202

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20100428

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20100511

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100802

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20100831

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20100930

FPAY Renewal fee payment (event date is renewal date of database)

Free format text: PAYMENT UNTIL: 20131008

Year of fee payment: 3

R150 Certificate of patent or registration of utility model

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

EXPY Cancellation because of completion of term