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)
- 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)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (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 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) | EP4389213A3 (es) |
| JP (4) | JP6752270B2 (es) |
| KR (3) | KR20240017126A (es) |
| CN (1) | CN109661230A (es) |
| AR (1) | AR109103A1 (es) |
| AU (2) | AU2017298376A1 (es) |
| CA (1) | CA3031249A1 (es) |
| CL (1) | CL2018001677A1 (es) |
| CO (1) | CO2018006358A2 (es) |
| IL (2) | IL313907A (es) |
| MX (2) | MX2018007669A (es) |
| TW (1) | TW202408513A (es) |
| WO (1) | WO2018017721A1 (es) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2533050B3 (en) | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | 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 |
| EP4389213A3 (en) * | 2016-07-19 | 2024-08-21 | 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 |
| DK4062916T3 (da) | 2017-05-30 | 2025-01-13 | Amicus Therapeutics Inc | Behandling af fabry-patienter med specifikke mutationer |
| WO2018222655A1 (en) | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| CA3090496C (en) * | 2018-02-06 | 2024-03-26 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| LT3749308T (lt) * | 2018-02-06 | 2024-02-12 | Amicus Therapeutics, Inc. | Klasikine fabri liga sergančių pacientų gydymas migalastatu |
| AU2019326252A1 (en) * | 2018-08-20 | 2021-02-25 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the GLA gene |
| US20220313670A1 (en) * | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| BR112022002202A2 (pt) | 2019-08-07 | 2022-06-14 | Amicus Therapeutics Inc | Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla |
| JP2023513700A (ja) * | 2020-02-10 | 2023-04-03 | アミカス セラピューティックス インコーポレイテッド | ファブリー病の治療方法 |
| 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 |
| EP0536402A4 (en) | 1990-06-29 | 1993-05-12 | Nippon Shinyaku Company, Limited | 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 |
| SI2444102T1 (sl) | 2003-01-31 | 2015-08-31 | Mount Sinai School Of Medicine Of New York University | Kombinirana terapija za zdravljenje motenj pomanjkanja proteina |
| US7446098B2 (en) | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US7741340B2 (en) | 2003-11-12 | 2010-06-22 | Amicus Therapeutics, Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
| AU2006247065B8 (en) | 2005-05-17 | 2012-07-12 | Amicus Therapeutics, Inc. | A method for the treatment of Pompe disease using 1-deoxynojirimycin 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 |
| JP2009502954A (ja) | 2005-07-27 | 2009-01-29 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド | タンパク質のミスフォールディングを修正する小分子及びその使用 |
| EP2533050B3 (en) * | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | Treatment Options For Fabry Disease |
| AU2015230773B2 (en) * | 2006-05-16 | 2017-09-28 | Amicus Therapeutics Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| EP2081904B1 (en) | 2006-09-22 | 2011-03-02 | Krka Tovarna Zdravil, D.D., Novo Mesto | Aripiprazole hemifumarate and process for its preparation |
| WO2008121826A2 (en) | 2007-03-30 | 2008-10-09 | 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 |
| SG10201604757RA (en) * | 2011-03-11 | 2016-08-30 | 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 |
| EP4389213A3 (en) * | 2016-07-19 | 2024-08-21 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naive and ert-experienced patients |
-
2017
- 2017-07-19 EP EP24168026.3A patent/EP4389213A3/en active Pending
- 2017-07-19 MX MX2018007669A patent/MX2018007669A/es unknown
- 2017-07-19 KR KR1020247003290A patent/KR20240017126A/ko 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 TW TW112140206A patent/TW202408513A/zh unknown
- 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 AU AU2017298376A patent/AU2017298376A1/en not_active Abandoned
- 2017-07-19 EP EP23219937.2A patent/EP4324521A3/en active Pending
- 2017-07-19 EP EP24168024.8A patent/EP4378524A3/en active Pending
- 2017-07-19 CN CN201780054621.XA patent/CN109661230A/zh active Pending
- 2017-07-19 KR KR1020237013747A patent/KR20230061563A/ko not_active Ceased
- 2017-07-19 IL IL313907A patent/IL313907A/en unknown
- 2017-07-19 WO PCT/US2017/042872 patent/WO2018017721A1/en active Application Filing
- 2017-07-19 KR KR1020197004780A patent/KR20190030729A/ko not_active Ceased
- 2017-07-19 EP EP23219947.1A patent/EP4356911A1/en active Pending
- 2017-07-19 AR ARP170102027A patent/AR109103A1/es unknown
-
2018
- 2018-06-20 CO CONC2018/0006358A patent/CO2018006358A2/es unknown
- 2018-06-20 CL CL2018001677A patent/CL2018001677A1/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/AU2023206175A1/en active Pending
- 2023-11-13 JP JP2023192778A patent/JP2024026079A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4389213A2 (en) | 2024-06-26 |
| WO2018017721A1 (en) | 2018-01-25 |
| US20230321065A1 (en) | 2023-10-12 |
| TW201806597A (zh) | 2018-03-01 |
| KR20230061563A (ko) | 2023-05-08 |
| AU2023206175A1 (en) | 2023-08-10 |
| EP4324521A2 (en) | 2024-02-21 |
| US20190183869A1 (en) | 2019-06-20 |
| CA3031249A1 (en) | 2018-01-25 |
| JP2019178140A (ja) | 2019-10-17 |
| MX2022011334A (es) | 2022-10-07 |
| KR20240017126A (ko) | 2024-02-06 |
| JP6752270B2 (ja) | 2020-09-09 |
| JP2022033749A (ja) | 2022-03-02 |
| AR109103A1 (es) | 2018-10-31 |
| KR20190030729A (ko) | 2019-03-22 |
| IL313907A (en) | 2024-08-01 |
| CN109661230A (zh) | 2019-04-19 |
| TW202408513A (zh) | 2024-03-01 |
| US12070453B2 (en) | 2024-08-27 |
| JP2018526368A (ja) | 2018-09-13 |
| EP3487502A1 (en) | 2019-05-29 |
| CO2018006358A2 (es) | 2018-11-30 |
| EP4356911A1 (en) | 2024-04-24 |
| EP4389213A3 (en) | 2024-08-21 |
| MX2018007669A (es) | 2018-09-12 |
| EP4324521A3 (en) | 2024-04-24 |
| EP4378524A2 (en) | 2024-06-05 |
| AU2017298376A1 (en) | 2019-03-07 |
| IL264301A (en) | 2019-02-28 |
| JP2024026079A (ja) | 2024-02-28 |
| EP4378524A3 (en) | 2024-08-14 |
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