CO2018006358A2 - 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
- CO2018006358A2 CO2018006358A2 CONC2018/0006358A CO2018006358A CO2018006358A2 CO 2018006358 A2 CO2018006358 A2 CO 2018006358A2 CO 2018006358 A CO2018006358 A CO 2018006358A CO 2018006358 A2 CO2018006358 A2 CO 2018006358A2
- Authority
- CO
- Colombia
- 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
- 239000012458 free base Substances 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
- 210000000557 podocyte Anatomy 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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Chemical & Material Sciences (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Hematology (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 (2)
| 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 |
| PCT/US2017/042872 WO2018017721A1 (en) | 2016-07-19 | 2017-07-19 | Treatment of fabry disease in ert-naïve and ert-experienced patients |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2018006358A2 true CO2018006358A2 (es) | 2018-11-30 |
Family
ID=59416847
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2018/0006358A CO2018006358A2 (es) | 2016-07-19 | 2018-06-20 | Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US12070453B2 (es) |
| EP (5) | EP4378524A3 (es) |
| JP (4) | JP6752270B2 (es) |
| KR (3) | KR20190030729A (es) |
| CN (1) | CN109661230A (es) |
| AR (1) | 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) |
| NZ (2) | NZ750752A (es) |
| TW (2) | TWI875676B (es) |
| WO (1) | WO2018017721A1 (es) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007137072A2 (en) | 2006-05-16 | 2007-11-29 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| AU2009214648B2 (en) | 2008-02-12 | 2014-11-13 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| 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 |
| US12020820B1 (en) * | 2017-03-03 | 2024-06-25 | Cerner Innovation, Inc. | Predicting sphingolipidoses (fabry's disease) and decision support |
| CA3224529A1 (en) | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| 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 |
| JP2021512896A (ja) * | 2018-02-06 | 2021-05-20 | アミカス セラピューティックス インコーポレイテッド | 古典型ファブリー病患者の治療 |
| CA3090496C (en) * | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| ES2993833T3 (en) * | 2018-08-20 | 2025-01-10 | Amicus Therapeutics Inc | Migalastat for use in 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 |
| US20220313670A1 (en) * | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| US11833164B2 (en) | 2019-08-07 | 2023-12-05 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| CA3167476A1 (en) * | 2020-02-10 | 2021-08-19 | Nina SKUBAN | Methods of treating fabry disease |
| 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 |
| WO1992000277A1 (en) | 1990-06-29 | 1992-01-09 | Nippon Shinyaku Co., Ltd. | Piperidine derivative |
| JP2000509971A (ja) | 1996-04-10 | 2000-08-08 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 化学的シャペローンを用いる遺伝的欠陥の修正 |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | 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 |
| 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 |
| CN1901907A (zh) | 2003-11-12 | 2007-01-24 | 阿米库斯治疗学公司 | 用于治疗戈谢病的葡糖并咪唑和多羟基环己烯基胺衍生物 |
| ES2572148T3 (es) | 2005-05-17 | 2016-05-30 | Amicus Therapeutics Inc | Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados |
| CN104771402A (zh) | 2005-06-08 | 2015-07-15 | 阿米库斯治疗学公司 | 溶酶体酶编码基因突变相关的cns紊乱的治疗 |
| US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
| EP1909812A4 (en) | 2005-07-27 | 2009-11-25 | Univ Florida | SMALL CONNECTIONS FOR THE CORRECTION OF PROTEIN MISFAIRINGS AND USES THEREOF |
| AU2015230773B2 (en) | 2006-05-16 | 2017-09-28 | Amicus Therapeutics Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| WO2007137072A2 (en) * | 2006-05-16 | 2007-11-29 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| UA94624C2 (uk) | 2006-09-22 | 2011-05-25 | Крка, Товарна Здравіл, Д.Д., Ново Мєсто | Арипіпразолу геміфумарат і спосіб його одержання |
| US20100113517A1 (en) | 2007-03-30 | 2010-05-06 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease using pharmacological chaperones |
| AU2009214648B2 (en) | 2008-02-12 | 2014-11-13 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| CN107088225A (zh) * | 2011-03-11 | 2017-08-25 | 阿米库斯治疗学公司 | 治疗法布里病的给药方案 |
| 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 |
| 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 |
-
2017
- 2017-07-19 TW TW106124148A patent/TWI875676B/zh active
- 2017-07-19 WO PCT/US2017/042872 patent/WO2018017721A1/en not_active Ceased
- 2017-07-19 IL IL313907A patent/IL313907A/en unknown
- 2017-07-19 EP EP24168024.8A patent/EP4378524A3/en active Pending
- 2017-07-19 EP EP23219947.1A patent/EP4356911A1/en active Pending
- 2017-07-19 EP EP24168026.3A patent/EP4389213A3/en active Pending
- 2017-07-19 CA CA3031249A patent/CA3031249A1/en active Pending
- 2017-07-19 JP JP2018510380A patent/JP6752270B2/ja active Active
- 2017-07-19 CN CN201780054621.XA patent/CN109661230A/zh active Pending
- 2017-07-19 MX MX2018007669A patent/MX395523B/es unknown
- 2017-07-19 AR ARP170102027A patent/AR109103A1/es not_active Application Discontinuation
- 2017-07-19 US US16/318,905 patent/US12070453B2/en active Active
- 2017-07-19 EP EP23219937.2A patent/EP4324521A3/en active Pending
- 2017-07-19 KR KR1020197004780A patent/KR20190030729A/ko not_active Ceased
- 2017-07-19 KR KR1020247003290A patent/KR20240017126A/ko not_active Ceased
- 2017-07-19 TW TW112140206A patent/TW202408513A/zh unknown
- 2017-07-19 KR KR1020237013747A patent/KR20230061563A/ko not_active Ceased
- 2017-07-19 AU AU2017298376A patent/AU2017298376A1/en not_active Abandoned
- 2017-07-19 NZ NZ750752A patent/NZ750752A/en unknown
- 2017-07-19 EP EP17745612.6A patent/EP3487502A1/en not_active Withdrawn
- 2017-07-19 NZ NZ790449A patent/NZ790449A/en unknown
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2018
- 2018-06-20 CL CL2018001677A patent/CL2018001677A1/es unknown
- 2018-06-20 CO CONC2018/0006358A patent/CO2018006358A2/es unknown
- 2018-06-20 MX MX2022011334A patent/MX2022011334A/es unknown
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2019
- 2019-01-17 IL IL264301A patent/IL264301A/en unknown
- 2019-05-22 JP JP2019096045A patent/JP2019178140A/ja not_active Withdrawn
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2021
- 2021-11-08 JP JP2021181583A patent/JP2022033749A/ja not_active Withdrawn
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2023
- 2023-05-18 US US18/199,120 patent/US20230321065A1/en active Pending
- 2023-07-20 AU AU2023206175A patent/AU2023206175B2/en active Active
- 2023-11-13 JP JP2023192778A patent/JP2024026079A/ja active Pending
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2025
- 2025-08-16 AU AU2025217396A patent/AU2025217396A1/en active Pending
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