AR109103A1 - 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
- AR109103A1 AR109103A1 ARP170102027A ARP170102027A AR109103A1 AR 109103 A1 AR109103 A1 AR 109103A1 AR P170102027 A ARP170102027 A AR P170102027A AR P170102027 A ARP170102027 A AR P170102027A AR 109103 A1 AR109103 A1 AR 109103A1
- Authority
- AR
- Argentina
- Prior art keywords
- treated
- fabry
- ert
- treatment
- disease
- Prior art date
Links
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)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Heart & Thoracic Surgery (AREA)
- Urology & Nephrology (AREA)
- Cardiology (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 (naïve) o tratados con ERT (terapia de reemplazo enzimático). 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. Reivindicación 1: Un método para reducir el índice de masa ventricular izquierda (LVMi) en un paciente tratado previamente con una terapia de reemplazo enzimático (ERT) que sufre de la enfermedad de Fabry, caracterizado porque dicho método comprende administrar al paciente una formulación que comprende una cantidad eficaz de migalastat o una sal del mismo cada dos días, en donde la cantidad eficaz es de aproximadamente 123 mg de equivalente de base libre (FBE).
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 |
|---|---|
| AR109103A1 true AR109103A1 (es) | 2018-10-31 |
Family
ID=59416847
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP170102027A AR109103A1 (es) | 2016-07-19 | 2017-07-19 | 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) | EP3487502A1 (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 (1) | NZ790449A (es) |
| TW (2) | TW202408513A (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 |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| EP3487502A1 (en) * | 2016-07-19 | 2019-05-29 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| US12020820B1 (en) * | 2017-03-03 | 2024-06-25 | Cerner Innovation, Inc. | Predicting sphingolipidoses (fabry's disease) and decision support |
| HUE065615T2 (hu) | 2017-05-30 | 2024-06-28 | Amicus Therapeutics Inc | Migalasztát, vesekárosodással szövõdött Fabry-kórban szenvedõ betegeg kezelésére |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| EP3749307B1 (en) * | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| RS65103B1 (sr) * | 2018-02-06 | 2024-02-29 | Amicus Therapeutics Inc | Lečenje bolesnika sa klasičnom fabrijevom bolešću migalastatom |
| 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 |
| BR112021024886A2 (pt) * | 2019-06-11 | 2022-01-25 | Amicus Therapeutics Inc | Métodos de tratar a doença de fabry em pacientes com insuficiência renal |
| JP2021024864A (ja) | 2019-08-07 | 2021-02-22 | アミカス セラピューティックス インコーポレイテッド | Ｇｌａ遺伝子に突然変異を有する患者のファブリー病を治療する方法 |
| AU2021218578A1 (en) * | 2020-02-10 | 2022-08-25 | Amicus Therapeutics, Inc. | 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 |
| CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
| EP0959877A4 (en) | 1996-04-10 | 2000-08-23 | Univ California | CORRECTION OF GENETIC ERRORS USING CHEMICAL COMPANIES |
| 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 |
| DK1589993T3 (en) | 2003-01-31 | 2015-03-09 | Sinai School Medicine | COMBINATION THERAPY FOR TREATMENT OF PROTEIN FAILURE DISORDERS |
| US7446098B2 (en) | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| EP1682134A4 (en) | 2003-11-12 | 2008-10-15 | Amicus Therapeutics Inc | GLUCOIMIDAZOLE AND POLYHYDROXYCYCLOHEXENYL-AMINE DERIVATIVES FOR THE TREATMENT OF GAUCHER DISEASE |
| CA2612538C (en) | 2005-05-17 | 2015-06-30 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| 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 |
| CN101267779A (zh) | 2005-07-27 | 2008-09-17 | 福罗里达大学研究基金会有限公司 | 使用热休克治疗眼睛疾病 |
| 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 |
| ATE500226T1 (de) | 2006-09-22 | 2011-03-15 | Krka Tovarna Zdravil D D Novo Mesto | Aripiprazolhemifumarat und verfahren zu dessen herstellung |
| US20100113517A1 (en) | 2007-03-30 | 2010-05-06 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease using pharmacological chaperones |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| SG193379A1 (en) * | 2011-03-11 | 2013-10-30 | 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 |
| EP3487502A1 (en) * | 2016-07-19 | 2019-05-29 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
-
2017
- 2017-07-19 EP EP17745612.6A patent/EP3487502A1/en not_active Withdrawn
- 2017-07-19 MX MX2018007669A patent/MX395523B/es unknown
- 2017-07-19 US US16/318,905 patent/US12070453B2/en active Active
- 2017-07-19 TW TW112140206A patent/TW202408513A/zh unknown
- 2017-07-19 TW TW106124148A patent/TWI875676B/zh active
- 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 EP EP23219937.2A patent/EP4324521A3/en active Pending
- 2017-07-19 NZ NZ790449A patent/NZ790449A/en unknown
- 2017-07-19 CN CN201780054621.XA patent/CN109661230A/zh active Pending
- 2017-07-19 CA CA3031249A patent/CA3031249A1/en active Pending
- 2017-07-19 IL IL313907A patent/IL313907A/en unknown
- 2017-07-19 JP JP2018510380A patent/JP6752270B2/ja active Active
- 2017-07-19 AR ARP170102027A patent/AR109103A1/es not_active Application Discontinuation
- 2017-07-19 AU AU2017298376A patent/AU2017298376A1/en not_active Abandoned
- 2017-07-19 KR KR1020197004780A patent/KR20190030729A/ko not_active Ceased
- 2017-07-19 KR KR1020237013747A patent/KR20230061563A/ko not_active Ceased
- 2017-07-19 KR KR1020247003290A patent/KR20240017126A/ko not_active Ceased
- 2017-07-19 WO PCT/US2017/042872 patent/WO2018017721A1/en not_active Ceased
-
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
-
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/US20230321065A1/en active Pending
- 2023-07-20 AU AU2023206175A patent/AU2023206175B2/en active Active
- 2023-11-13 JP JP2023192778A patent/JP2024026079A/ja active Pending
-
2025
- 2025-08-16 AU AU2025217396A patent/AU2025217396A1/en active Pending
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Legal Events
| Date | Code | Title | Description |
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| FC | Refusal |