CL2018001677A1 - Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert - Google Patents
Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ertInfo
- Publication number
- CL2018001677A1 CL2018001677A1 CL2018001677A CL2018001677A CL2018001677A1 CL 2018001677 A1 CL2018001677 A1 CL 2018001677A1 CL 2018001677 A CL2018001677 A CL 2018001677A CL 2018001677 A CL2018001677 A CL 2018001677A CL 2018001677 A1 CL2018001677 A1 CL 2018001677A1
- Authority
- CL
- Chile
- Prior art keywords
- treated
- treatment
- ert
- patients
- fabry disease
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 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
- 230000002861 ventricular Effects 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
- A61K31/33—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
- A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/02—Antidotes
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Urology & Nephrology (AREA)
- Toxicology (AREA)
- Molecular Biology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
Abstract
SE PROVEEN REGÍMENES DE DOSIFICACIÓN PARA EL TRATAMIENTO DE LA ENFERMEDAD DE FABRY EN UN PACIENTE. ALGUNOS MÉTODOS SE RELACIONAN CON EL TRATAMIENTO DE PACIENTES CON FABRY NO TRATADOS PREVIAMENTE O TRATADOS CON ERT. ALGUNOS MÉTODOS COMPRENDEN ADMINISTRAR AL PACIENTE APROXIMADAMENTE 123 MG DE EQUIVALENTES DE BASE LIBRE DE MIGALASTAT PARA MEJORAR LA MASA VENTRICULAR IZQUIERDA Y/O PARA MEJORAR LA GLOBOTRIAOSILCERAMIDA EN PODOCITOS.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15/213,920 US9999618B2 (en) | 2007-04-26 | 2016-07-19 | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2018001677A1 true CL2018001677A1 (es) | 2019-02-01 |
Family
ID=59416847
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2018001677A CL2018001677A1 (es) | 2016-07-19 | 2018-06-20 | Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US12070453B2 (es) |
| EP (5) | EP3487502A1 (es) |
| JP (4) | JP6752270B2 (es) |
| KR (3) | KR20240017126A (es) |
| CN (1) | CN109661230A (es) |
| AR (2) | AR109103A1 (es) |
| AU (3) | AU2017298376A1 (es) |
| CA (1) | CA3031249A1 (es) |
| CL (1) | CL2018001677A1 (es) |
| CO (1) | CO2018006358A2 (es) |
| IL (2) | IL313907A (es) |
| MX (2) | MX395523B (es) |
| TW (2) | TWI875676B (es) |
| WO (1) | WO2018017721A1 (es) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009537149A (ja) | 2006-05-16 | 2009-10-29 | 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 | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| AU2017298376A1 (en) * | 2016-07-19 | 2019-03-07 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naive and ERT-experienced patients |
| US12020820B1 (en) * | 2017-03-03 | 2024-06-25 | Cerner Innovation, Inc. | Predicting sphingolipidoses (fabry's disease) and decision support |
| PT4324522T (pt) | 2017-05-30 | 2026-03-13 | Amicus Therapeutics Inc | Métodos de tratamento de pacientes com fabry tendo insuficiência renal |
| KR20220116305A (ko) | 2017-05-30 | 2022-08-22 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| PT3749308T (pt) * | 2018-02-06 | 2024-01-25 | Amicus Therapeutics Inc | Tratamento de pacientes com doença de fabry clássica com migalastato |
| EP3749307B1 (en) * | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| EP4289476A3 (en) * | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| TW202042812A (zh) * | 2019-01-22 | 2020-12-01 | 美商阿米庫斯醫療股份有限公司 | 減少法布瑞氏症患者中之腦血管事件之方法 |
| BR112021024886A2 (pt) * | 2019-06-11 | 2022-01-25 | Amicus Therapeutics Inc | Métodos de tratar a doença de fabry em pacientes com insuficiência renal |
| WO2021026447A1 (en) | 2019-08-07 | 2021-02-11 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| PL4103178T3 (pl) * | 2020-02-10 | 2026-05-11 | Amicus Therapeutics, Inc. | Sposoby leczenia choroby fabry'ego |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| KR102471225B1 (ko) | 2021-11-18 | 2022-11-28 | 박기용 | 어플리케이션을 통한 소비자 및 농장 연결 시스템 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0463109A4 (en) | 1989-03-24 | 1992-11-19 | Research Corporation Technologies, Inc. | Recombinant alpha-galactosidase, a therapy for fabry disease |
| CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
| US5900360A (en) | 1996-04-10 | 1999-05-04 | Welch; William J. | Correction of genetic defects using chemical chaperones |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| PT2857036T (pt) | 2003-01-31 | 2017-03-13 | Mount Sinai School Of Medicine Of N Y Univ | 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 |
| 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 |
| AU2006247065B8 (en) | 2005-05-17 | 2012-07-12 | Amicus Therapeutics, Inc. | A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives |
| 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. |
| US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
| CA2616537A1 (en) | 2005-07-27 | 2007-02-01 | University Of Florida Research Foundation, Inc. | Small compounds that correct protein misfolding and uses thereof |
| AU2015230773B2 (en) | 2006-05-16 | 2017-09-28 | Amicus Therapeutics Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| JP2009537149A (ja) * | 2006-05-16 | 2009-10-29 | 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 | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| UA94624C2 (uk) | 2006-09-22 | 2011-05-25 | Крка, Товарна Здравіл, Д.Д., Ново Мєсто | Арипіпразолу геміфумарат і спосіб його одержання |
| MX2009010557A (es) | 2007-03-30 | 2009-11-19 | Amicus Therapeutics Inc | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| US20140219986A1 (en) * | 2011-03-11 | 2014-08-07 | Amicus Therapeutics ,Inc. | Dosing regimens for the treatment of fabry disease |
| JP2013255488A (ja) | 2012-05-18 | 2013-12-26 | Saitama Medical Univ | 拡張期心不全非ヒトモデル動物及びその製造方法、並びに、拡張期心不全予防乃至症状改善活性を有する物質のスクリーニング方法 |
| AU2017298376A1 (en) * | 2016-07-19 | 2019-03-07 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naive and ERT-experienced patients |
-
2017
- 2017-07-19 AU AU2017298376A patent/AU2017298376A1/en not_active Abandoned
- 2017-07-19 US US16/318,905 patent/US12070453B2/en active Active
- 2017-07-19 EP EP17745612.6A patent/EP3487502A1/en not_active Withdrawn
- 2017-07-19 TW TW106124148A patent/TWI875676B/zh active
- 2017-07-19 KR KR1020247003290A patent/KR20240017126A/ko active Pending
- 2017-07-19 CN CN201780054621.XA patent/CN109661230A/zh active Pending
- 2017-07-19 EP EP24168026.3A patent/EP4389213A3/en active Pending
- 2017-07-19 KR KR1020237013747A patent/KR20230061563A/ko not_active Ceased
- 2017-07-19 EP EP23219947.1A patent/EP4356911B1/en active Active
- 2017-07-19 CA CA3031249A patent/CA3031249A1/en active Pending
- 2017-07-19 EP EP23219937.2A patent/EP4324521B1/en active Active
- 2017-07-19 EP EP24168024.8A patent/EP4378524A3/en active Pending
- 2017-07-19 MX MX2018007669A patent/MX395523B/es unknown
- 2017-07-19 IL IL313907A patent/IL313907A/en unknown
- 2017-07-19 KR KR1020197004780A patent/KR20190030729A/ko not_active Ceased
- 2017-07-19 JP JP2018510380A patent/JP6752270B2/ja active Active
- 2017-07-19 TW TW112140206A patent/TW202408513A/zh unknown
- 2017-07-19 WO PCT/US2017/042872 patent/WO2018017721A1/en not_active Ceased
- 2017-07-19 AR ARP170102027A patent/AR109103A1/es not_active Application Discontinuation
-
2018
- 2018-06-20 MX MX2022011334A patent/MX2022011334A/es unknown
- 2018-06-20 CO CONC2018/0006358A patent/CO2018006358A2/es unknown
- 2018-06-20 CL CL2018001677A patent/CL2018001677A1/es unknown
-
2019
- 2019-01-17 IL IL264301A patent/IL264301A/en unknown
- 2019-05-22 JP JP2019096045A patent/JP2019178140A/ja not_active Withdrawn
-
2021
- 2021-11-08 JP JP2021181583A patent/JP2022033749A/ja not_active Withdrawn
-
2023
- 2023-05-18 US US18/199,120 patent/US12599594B2/en active Active
- 2023-07-20 AU AU2023206175A patent/AU2023206175B2/en active Active
- 2023-11-13 JP JP2023192778A patent/JP2024026079A/ja active Pending
-
2025
- 2025-01-03 AR ARP250100029A patent/AR134835A2/es unknown
- 2025-08-16 AU AU2025217396A patent/AU2025217396A1/en active Pending
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