AR114392A1 - Tratamiento de pacientes con enfermedad de fabry clásica - Google Patents
Tratamiento de pacientes con enfermedad de fabry clásicaInfo
- Publication number
- AR114392A1 AR114392A1 ARP190100287A ARP190100287A AR114392A1 AR 114392 A1 AR114392 A1 AR 114392A1 AR P190100287 A ARP190100287 A AR P190100287A AR P190100287 A ARP190100287 A AR P190100287A AR 114392 A1 AR114392 A1 AR 114392A1
- Authority
- AR
- Argentina
- Prior art keywords
- patient
- renal
- fabry disease
- reduce
- patients
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/20—Pills, tablets, discs, rods
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Urology & Nephrology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (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)
Abstract
Se proveen métodos para el tratamiento de la enfermedad de Fabry clásica en un paciente. Algunos métodos comprenden administrar al paciente aproximadamente 123 mg equivalentes de base libre de migalastat para reducir la globotriaosilceramida renal, estabilizar la función renal, reducir la masa ventricular izquierda, reducir la globotriaosisesfingosina en plasma y/o tratar síntomas gastrointestinales. Reivindicación 1: Un método para reducir la globotriaosilceramida renal (GL-3) en un paciente que tiene enfermedad de Fabry clásica, comprendiendo el método administrar al paciente una cantidad eficaz de migalastat o sal del mismo a una frecuencia de una vez cada dos días para reducir la GL-3 renal del paciente, en donde la cantidad eficaz es de aproximadamente 100 mg a aproximadamente 150 mg equivalentes de base libre (FBE).
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862626992P | 2018-02-06 | 2018-02-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR114392A1 true AR114392A1 (es) | 2020-09-02 |
Family
ID=65494622
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190100287A AR114392A1 (es) | 2018-02-06 | 2019-02-06 | Tratamiento de pacientes con enfermedad de fabry clásica |
Country Status (20)
| Country | Link |
|---|---|
| US (1) | US20210038579A1 (es) |
| EP (2) | EP3749308B1 (es) |
| JP (2) | JP2021512896A (es) |
| KR (1) | KR20200128675A (es) |
| CN (1) | CN112203657A (es) |
| AR (1) | AR114392A1 (es) |
| AU (1) | AU2019217603A1 (es) |
| CA (1) | CA3090499A1 (es) |
| DK (1) | DK3749308T3 (es) |
| ES (1) | ES2969263T3 (es) |
| FI (1) | FI3749308T3 (es) |
| HR (1) | HRP20240025T1 (es) |
| HU (1) | HUE064761T2 (es) |
| LT (1) | LT3749308T (es) |
| PL (1) | PL3749308T3 (es) |
| PT (1) | PT3749308T (es) |
| RS (1) | RS65103B1 (es) |
| SI (1) | SI3749308T1 (es) |
| TW (1) | TW201944998A (es) |
| WO (1) | WO2019157056A1 (es) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3457135A1 (en) | 2006-05-16 | 2019-03-20 | Amicus Therapeutics, Inc. | Treatment options for fabry disease |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| KR20240017112A (ko) | 2017-05-30 | 2024-02-06 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| EP3749307A1 (en) | 2018-02-06 | 2020-12-16 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| CA3147055A1 (en) | 2019-08-07 | 2021-02-11 | Elfrida Benjamin | Methods of treating fabry disease in patients having a mutation in the gla gene |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (7)
| 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 |
| EP3457135A1 (en) * | 2006-05-16 | 2019-03-20 | Amicus Therapeutics, Inc. | Treatment options for fabry disease |
| MX2009010557A (es) * | 2007-03-30 | 2009-11-19 | Amicus Therapeutics Inc | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| CN114159436A (zh) * | 2016-03-22 | 2022-03-11 | 阿米库斯治疗学公司 | 治疗在gla基因中具有g9331a突变的患者的法布里病的方法 |
| US20190183869A1 (en) * | 2016-07-19 | 2019-06-20 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| SG11202000423XA (en) * | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
-
2019
- 2019-02-06 US US16/967,824 patent/US20210038579A1/en active Pending
- 2019-02-06 EP EP19706182.3A patent/EP3749308B1/en active Active
- 2019-02-06 PT PT197061823T patent/PT3749308T/pt unknown
- 2019-02-06 DK DK19706182.3T patent/DK3749308T3/da active
- 2019-02-06 CN CN201980024335.8A patent/CN112203657A/zh active Pending
- 2019-02-06 EP EP23200414.3A patent/EP4316589A3/en active Pending
- 2019-02-06 RS RS20240081A patent/RS65103B1/sr unknown
- 2019-02-06 HU HUE19706182A patent/HUE064761T2/hu unknown
- 2019-02-06 CA CA3090499A patent/CA3090499A1/en active Pending
- 2019-02-06 HR HRP20240025TT patent/HRP20240025T1/hr unknown
- 2019-02-06 PL PL19706182.3T patent/PL3749308T3/pl unknown
- 2019-02-06 AR ARP190100287A patent/AR114392A1/es unknown
- 2019-02-06 JP JP2020542569A patent/JP2021512896A/ja active Pending
- 2019-02-06 ES ES19706182T patent/ES2969263T3/es active Active
- 2019-02-06 AU AU2019217603A patent/AU2019217603A1/en active Pending
- 2019-02-06 SI SI201930691T patent/SI3749308T1/sl unknown
- 2019-02-06 FI FIEP19706182.3T patent/FI3749308T3/fi active
- 2019-02-06 LT LTEPPCT/US2019/016853T patent/LT3749308T/lt unknown
- 2019-02-06 WO PCT/US2019/016853 patent/WO2019157056A1/en active Application Filing
- 2019-02-06 KR KR1020207025688A patent/KR20200128675A/ko unknown
- 2019-02-11 TW TW108104436A patent/TW201944998A/zh unknown
-
2023
- 2023-11-13 JP JP2023192942A patent/JP2024026080A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4316589A2 (en) | 2024-02-07 |
| KR20200128675A (ko) | 2020-11-16 |
| WO2019157056A8 (en) | 2020-12-03 |
| JP2024026080A (ja) | 2024-02-28 |
| CN112203657A (zh) | 2021-01-08 |
| ES2969263T3 (es) | 2024-05-17 |
| AU2019217603A1 (en) | 2020-09-24 |
| HRP20240025T1 (hr) | 2024-03-29 |
| EP3749308B1 (en) | 2023-10-25 |
| PL3749308T3 (pl) | 2024-03-25 |
| WO2019157056A1 (en) | 2019-08-15 |
| RS65103B1 (sr) | 2024-02-29 |
| SI3749308T1 (sl) | 2024-03-29 |
| EP3749308A1 (en) | 2020-12-16 |
| CA3090499A1 (en) | 2019-08-15 |
| PT3749308T (pt) | 2024-01-25 |
| TW201944998A (zh) | 2019-12-01 |
| EP4316589A3 (en) | 2024-04-24 |
| FI3749308T3 (fi) | 2024-01-18 |
| HUE064761T2 (hu) | 2024-04-28 |
| US20210038579A1 (en) | 2021-02-11 |
| LT3749308T (lt) | 2024-02-12 |
| DK3749308T3 (da) | 2024-01-22 |
| JP2021512896A (ja) | 2021-05-20 |
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