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 AInfo
- 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
Links
- 230000002132 lysosomal effect Effects 0.000 title abstract 2
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 102000005840 alpha-Galactosidase Human genes 0.000 title 1
- 108010030291 alpha-Galactosidase Proteins 0.000 title 1
- 208000024720 Fabry Disease Diseases 0.000 abstract 1
- 150000001875 compounds Chemical class 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000002708 enhancing effect Effects 0.000 abstract 1
- 210000004962 mammalian cell Anatomy 0.000 abstract 1
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 abstract 1
- 229950007469 migalastat Drugs 0.000 abstract 1
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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Vascular Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pyrrole Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
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 |
|---|---|---|---|
| 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 |
| 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 |
Family Applications Before (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) | DK2143420T3 (da) |
| ES (2) | ES2333561T3 (da) |
| PT (2) | PT2143420E (da) |
| WO (1) | WO1999062517A1 (da) |
Families Citing this family (105)
| 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 |
| US6849257B2 (en) | 2000-02-04 | 2005-02-01 | 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 |
| AU2003284886A1 (en) * | 2002-10-21 | 2004-05-13 | William E. Balch | CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE |
| EP2857036B1 (en) * | 2003-01-31 | 2016-12-21 | 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 |
| 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 |
| CA2545433A1 (en) * | 2003-11-12 | 2005-05-26 | 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 |
| US7488721B2 (en) * | 2004-05-13 | 2009-02-10 | Mcmaster University | Real time methylumbelliferone-based assay |
| US20090239807A1 (en) * | 2004-05-24 | 2009-09-24 | Mia Horowitz | Methods and kits for diagnosing and/or assessing severity and treating gaucher disease |
| JP2008503590A (ja) * | 2004-06-21 | 2008-02-07 | メドトロニック・インコーポレーテッド | 組成物を細胞にデリバリーするための医学用システム及び方法 |
| US8889127B2 (en) * | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
| FR2873377B1 (fr) * | 2004-07-23 | 2006-10-13 | Sanofi Synthelabo | Derives d'azasucre, inhibiteurs d'heparanases, leur procede de preparation, les compositions en contenant et leur utilisation |
| 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 |
| 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 |
| US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens 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 |
| PL2533050T6 (pl) | 2006-05-16 | 2016-05-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry'ego |
| AU2013205532B8 (en) * | 2006-05-16 | 2015-07-16 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| PL1860101T3 (pl) * | 2006-05-24 | 2011-10-31 | Amicus Therapeutics Inc | Sól winianu izofagominy i sposoby zastosowania |
| ES2364586T3 (es) * | 2006-05-24 | 2011-09-07 | Amicus Therapeutics, Inc. | Sal tartrato de isofagomina y métodos de uso. |
| WO2007150064A2 (en) | 2006-06-23 | 2007-12-27 | Amicus Therapeutics, Inc. | METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-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 |
| WO2008075957A1 (en) * | 2006-12-21 | 2008-06-26 | 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 |
| MX2009010557A (es) * | 2007-03-30 | 2009-11-19 | Amicus Therapeutics Inc | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
| 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 |
| AU2008240205A1 (en) * | 2007-04-13 | 2008-10-23 | Amicus Therapeutics, Inc. | Use of substrates as 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. |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| CA2688850C (en) | 2007-05-22 | 2015-08-11 | 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 |
| EP2252288A1 (en) * | 2007-11-21 | 2010-11-24 | Summit Corporation Plc | Treatment of protein folding disorders |
| EP2946785B1 (en) * | 2008-02-12 | 2018-10-24 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| KR20100127842A (ko) * | 2008-03-26 | 2010-12-06 | 유니버시티 오브 옥스퍼드 | 소포체 표적화 리포좀 |
| US8540985B2 (en) | 2008-06-26 | 2013-09-24 | 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 |
| 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 |
| 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 |
| 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 |
| US9044437B2 (en) | 2009-10-19 | 2015-06-02 | Amicus Therapeutics, Inc. | Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases |
| SI2995306T1 (sl) | 2009-10-19 | 2019-04-30 | Amicus Therapeutics, Inc. | Novi sestavki za preprečevanje in/ali zdravljenje motenj lizosomskega shranjevanja |
| KR101880411B1 (ko) | 2009-10-19 | 2018-07-19 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물 |
| US9194011B2 (en) | 2009-11-17 | 2015-11-24 | Protalix Ltd. | Stabilized alpha-galactosidase and uses thereof |
| KR101784539B1 (ko) * | 2010-01-28 | 2017-10-11 | 랩터 파마슈티컬스 인코포레이티드 | 수용체 결합 단백질(rap) 펩타이드-푸코시다제 억제제 접합체를 이용한 간 질환의 치료방법 |
| WO2011095893A1 (en) * | 2010-02-02 | 2011-08-11 | University Of Oxford | Synthesis of dgjnac from d-glucuronolactone and use to inhibit alpha-n-acetylgalactosaminidases |
| RU2013125923A (ru) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| SG10201604757RA (en) * | 2011-03-11 | 2016-08-30 | Amicus Therapeutics Inc | Dosing regimens for the treatment of fabry disease |
| LT2753346T (lt) | 2011-09-07 | 2020-08-10 | Mount Sinai School Of Medicine | Ceramidazė ir ląstelių diferenciacija |
| 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 |
| PT2968479T (pt) | 2013-03-14 | 2019-08-07 | Icahn School Med Mount Sinai | Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização |
| US9675627B2 (en) | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| US10709700B2 (en) | 2014-09-15 | 2020-07-14 | Orphazyme A/S | Arimoclomol formulation |
| CN114540327B (zh) | 2014-09-30 | 2026-03-24 | 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 |
| KR20250007693A (ko) | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| KR20190087673A (ko) * | 2016-03-22 | 2019-07-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법 |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| KR20210059796A (ko) | 2016-04-29 | 2021-05-25 | 오르파짐 에이/에스 | 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰 |
| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 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. |
| SMT202300187T1 (it) | 2017-05-15 | 2023-07-20 | Amicus Therapeutics Inc | Alfa-glucosidasi acida umana ricombinante |
| RS65230B1 (sr) | 2017-05-30 | 2024-03-29 | Amicus Therapeutics Inc | Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega |
| CA3224546C (en) | 2017-05-30 | 2026-02-24 | Amicus Therapeutics, Inc. | Use of migalastat in the treatment of fabry patients |
| PL3630725T3 (pl) | 2017-06-01 | 2026-03-23 | Idorsia Pharmaceuticals Ltd | Postać krystaliczna n-butylodeoksygalaktonojirymycyny |
| 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 |
| US20200222377A1 (en) | 2017-08-28 | 2020-07-16 | Amicus Therapeutics, Inc. | Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease |
| JP7252211B2 (ja) * | 2017-09-08 | 2023-04-04 | ザ ヌムール ファウンデーション | リソソーム蓄積症を治療するための薬剤、デバイス、および血液循環システム、ならびにリソソーム蓄積症を治療するための方法 |
| US11337802B2 (en) | 2017-09-19 | 2022-05-24 | Cardiovalve Ltd. | Heart valve delivery systems and methods |
| 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 |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| PT3749308T (pt) * | 2018-02-06 | 2024-01-25 | Amicus Therapeutics Inc | Tratamento de pacientes com doença de fabry clássica com migalastato |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| US12594268B2 (en) | 2018-08-20 | 2026-04-07 | 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 |
| MX2021015352A (es) | 2019-06-11 | 2022-04-06 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal. |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| JP2022548162A (ja) * | 2019-10-04 | 2022-11-16 | アカデミア シニカ | ポンペ病を処置する方法 |
| MX2022006366A (es) * | 2019-11-27 | 2022-06-22 | Turning Point Therapeutics Inc | Terapia de combinacion que implica compuestos de diarilo macrociclico. |
| KR20220150902A (ko) * | 2020-02-10 | 2022-11-11 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리 질병을 치료하는 방법 |
| 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 |
| FR3109523B1 (fr) * | 2020-04-23 | 2023-11-17 | Univ Grenoble Alpes | Nouveaux composés chaperons pharmacologiques de l’alpha-glucosidase acide humaine et leur utilisation thérapeutique |
| KR20230128462A (ko) | 2020-11-19 | 2023-09-05 | 제브라 덴마크 에이/에스 | 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| CA3225511A1 (en) | 2021-07-12 | 2023-01-19 | Franklin Johnson | Methods of treating fabry disease in pediatric patients |
| 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)
| 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 |
| CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
| US5276120A (en) | 1991-05-30 | 1994-01-04 | The Scripps Research Institute | Process for forming omega-deuxy-azasugars |
| US5399567A (en) * | 1993-05-13 | 1995-03-21 | Monsanto Company | Method of treating cholera |
| US6291657B1 (en) * | 1993-05-13 | 2001-09-18 | Monsanto Company | Deoxygalactonojirimycin derivatives |
| US5798366A (en) * | 1993-05-13 | 1998-08-25 | Monsanto Company | Method for treatment of CNS-involved lysosomal storage diseases |
| JPH0748398A (ja) | 1993-08-03 | 1995-02-21 | Nippon Oil Co Ltd | 変性蛋白質の再生方法及び変性蛋白質再生剤 |
| US5691306A (en) | 1993-08-26 | 1997-11-25 | National Research Council Of Canada | Methods of detection and treatment of protein trafficking disorders and increasing secretory protein production |
| 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 |
| 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 |
| ATE322900T1 (de) | 1996-07-15 | 2006-04-15 | Macrozyme Dnm B V | Deoxynojirimycin derivate und ihre verwendung als glukosesylceramidase-inhibitoren |
| MX340738B (es) | 1996-09-13 | 2016-07-22 | Shire Human Genetic Therapies | Terapia para deficiencia de a-galactosidasa 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 |
| PT1030839E (pt) | 1997-11-10 | 2004-05-31 | Searle & Co | Utilizacao de iminoacucares para tratar a resistencia a multifarmacos |
| US6465488B1 (en) | 1997-12-11 | 2002-10-15 | Chancellor, Masters & Scholars Of The University Of Oxford | Inhibition of glycolipid biosynthesis |
| WO1999040435A1 (en) | 1998-02-09 | 1999-08-12 | Netzer William J | Protein folding inhibitors |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| WO2000029556A2 (en) | 1998-11-17 | 2000-05-25 | The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services | Identification of glycosphingolipids that promote hiv-1 entry into cells |
| CN1329493A (zh) | 1998-12-02 | 2002-01-02 | 辉瑞产品公司 | 恢复p53家族蛋白质的构象稳定性的方法和组合物 |
| 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 |
| CA2306039A1 (en) | 1999-04-19 | 2000-10-19 | Schering Corporation | Hcv combination therapy |
| 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 & 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 |
| ES2302697T3 (es) | 1999-08-10 | 2008-08-01 | The Chancellor, Masters And Scholars Of The University Of Oxford | Compuestos n-alquilicos de cadena larga y derivados oxa de los mismos y uso como composiciones antivirales. |
| 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 |
| AU2001227972A1 (en) | 2000-01-20 | 2001-07-31 | Washington University | Methods to treat alpha-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 |
| WO2002028348A2 (en) | 2000-10-04 | 2002-04-11 | The Children's Hospital Of Philadelphia | Compositions and methods for treatment of cystic fibrosis |
| JP2002190202A (ja) | 2000-10-12 | 2002-07-05 | Koito Mfg Co Ltd | ヘッドランプ |
| JP3589449B2 (ja) | 2001-02-26 | 2004-11-17 | 株式会社ユニレック | 静電容量センサ、管内流動判断装置、管内流動制御装置 |
| JP4802548B2 (ja) * | 2005-04-28 | 2011-10-26 | シンフォニアテクノロジー株式会社 | Icチップ実装体の製造装置 |
| US7174582B1 (en) * | 2005-08-17 | 2007-02-13 | Kevin J Leary | Crib mattress bouncer |
| 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 |
| CA2787339A1 (en) | 2009-01-20 | 2011-08-12 | Signum Biosciences, Inc. | Anti-inflammatory complexes |
-
1998
- 1998-06-01 US US09/087,804 patent/US6274597B1/en not_active Expired - Lifetime
-
1999
- 1999-05-28 DK DK09011761.5T patent/DK2143420T3/da active
- 1999-05-28 AT AT99925611T patent/ATE442851T1/de active
- 1999-05-28 ES ES99925611T patent/ES2333561T3/es not_active Expired - Lifetime
- 1999-05-28 ES ES09011761.5T patent/ES2507515T3/es not_active Expired - Lifetime
- 1999-05-28 EP EP99925611A patent/EP1083899B1/en not_active Expired - Lifetime
- 1999-05-28 CA CA2333965A patent/CA2333965C/en not_active Expired - Lifetime
- 1999-05-28 WO PCT/US1999/010479 patent/WO1999062517A1/en not_active Ceased
- 1999-05-28 PT PT90117615T patent/PT2143420E/pt unknown
- 1999-05-28 AU AU41861/99A patent/AU4186199A/en not_active Abandoned
- 1999-05-28 PT PT99925611T patent/PT1083899E/pt unknown
- 1999-05-28 JP JP2000551773A patent/JP4602548B2/ja not_active Expired - Lifetime
- 1999-05-28 DE DE69941429T patent/DE69941429D1/de not_active Expired - Lifetime
- 1999-05-28 DK DK99925611.8T patent/DK1083899T3/da active
- 1999-05-28 EP EP09011761.5A patent/EP2143420B1/en not_active Expired - Lifetime
-
2000
- 2000-06-26 US US09/604,053 patent/US6583158B1/en not_active Expired - Lifetime
-
2001
- 2001-08-10 US US09/927,285 patent/US6774135B2/en not_active Expired - Lifetime
- 2001-09-07 US US09/948,348 patent/US6599919B2/en not_active Expired - Lifetime
-
2002
- 2002-06-14 US US10/172,604 patent/US6589964B2/en not_active Expired - Lifetime
- 2002-11-26 US US10/304,395 patent/US6916829B2/en not_active Expired - Lifetime
-
2004
- 2004-06-14 US US10/868,133 patent/US7622485B2/en not_active Expired - Fee Related
- 2004-11-16 US US10/989,258 patent/US7141582B2/en not_active Expired - Lifetime
-
2005
- 2005-10-31 US US11/264,672 patent/US7514453B2/en not_active Expired - Fee Related
-
2006
- 2006-09-27 US US11/528,903 patent/US7812033B2/en not_active Expired - Fee Related
-
2009
- 2009-11-23 US US12/624,026 patent/US8633221B2/en not_active Expired - Fee Related
- 2009-12-16 CY CY20091101305T patent/CY1112845T1/el unknown
-
2010
- 2010-08-02 JP JP2010173733A patent/JP5528941B2/ja not_active Expired - Lifetime
- 2010-09-24 US US12/890,014 patent/US8436018B2/en not_active Expired - Fee Related
-
2013
- 2013-04-22 US US13/867,914 patent/US8841322B2/en not_active Expired - Fee Related
- 2013-12-20 US US14/136,418 patent/US9265780B2/en not_active Expired - Fee Related
-
2014
- 2014-08-18 US US14/461,827 patent/US20150258129A1/en not_active Abandoned
- 2014-09-24 CY CY20141100776T patent/CY1115583T1/el unknown
-
2016
- 2016-01-25 US US15/005,371 patent/US20160346262A1/en not_active Abandoned
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK1083899T3 (da) | Derivater af 1-dexoy-galactonojirimycin og anvendelse heraf i behandlingen af lysosomale oplagringssygdomme ved øgning af lysosomal alpha-galactosidase A | |
| ES2115163T3 (es) | Procedimiento para inhibir la proliferacion de celulas de musculos lisos y la restinosis. | |
| DK125796A (da) | I form af små molekyler foreliggende inhibitorer for rotamaseaktivitet | |
| IL142824A0 (en) | Lipoic acid derivatives and their use in treatment of disease | |
| DK1228032T3 (da) | Præparater, som indeholder hydroxyicosatetraensyrederivater, og fremgangsmåder til anvendelse ved behandling af öjentörhedslidelser | |
| ATE261972T1 (de) | 2,5-diazabicyclo(2.2.1)heptanderivate, deren herstellung und deren verwendung in heilkunde | |
| DK0774005T3 (da) | Overfladeudtrykkelse af enzymer i genorienteret prodrug-terapi | |
| FI963221A0 (fi) | 6-substituoitu mykofenolihappo ja sen johdannaiset, joilla on immunosuppressiivista aktiivisuutta | |
| NO931534L (no) | Sulfonylbenzylsubstituerte benzo- og pyridopyridoner | |
| PT80820B (pt) | Processo para a preparacao de derivados de 1,3-propilidenodifosfonato 2-substituidos | |
| ATE531376T1 (de) | Verwendung von thioarabinofuranosyl-verbindungen | |
| NO303641B1 (no) | Optisk aktive benzotiepinderivater, mellomprodukter for deres fremstilling og deres anvendelse | |
| DE69534632T2 (de) | Clotrimazol-Metaboliten bei der Behandlung von Sichelzellenkrankheit | |
| NO970086D0 (no) | Heterocykliske azolonderivater med anti-Helicobacteraktivitet | |
| PT783495E (pt) | Derivados de quinoxalina uteis em terapia | |
| MY103210A (en) | Arylpiperazinyl-alkylene-phenyl-heterocyclic compounds. | |
| DE69414194D1 (de) | Aminosäurederivate und ihre Verwendung als Enkephalinase-Inhibitoren | |
| ITTO20000745A0 (it) | Nuovi acidi naftilsolfonici e composti ad essi correlati utli come agonisti dell'assorbimento del glucosio. | |
| DE69018099D1 (de) | Polyamin-Derivate als antineoplastische Mittel. | |
| CO5021190A1 (es) | Agentes terapeuticos | |
| DE69110861D1 (de) | Mittel zur verbesserung des kollagenmetabolismus und dessen verwendung. | |
| TR200003084T2 (tr) | Paroxetine askorbat | |
| NO944722L (no) | 3-(fenylalkylaminoalkyloksy)-5-fenylpyrazol-forbindelser samt fremgangsmåter og mellomprodukter for deres fremstilling, og legemidler som inneholder disse forbindelsene | |
| DK0791355T3 (da) | Fremgangsmåde til reduktion af vævsbeskadigelse forbundet med iskæmi | |
| VOSKANIAN et al. | The relationship between the radioprotective effect of helium-neon laser radiation in irradiated bacterial cells and the time interval between two types of irradiation(Zavisimost' radiozashchitnogo deistviia gelii-neonovogo lazernogo izlucheniia na kletki bakterii ot intervala vremeni mezhdu dvumia vidami oblucheniia) |