MX2009010557A - Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. - Google Patents
Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.Info
- Publication number
- MX2009010557A MX2009010557A MX2009010557A MX2009010557A MX2009010557A MX 2009010557 A MX2009010557 A MX 2009010557A MX 2009010557 A MX2009010557 A MX 2009010557A MX 2009010557 A MX2009010557 A MX 2009010557A MX 2009010557 A MX2009010557 A MX 2009010557A
- Authority
- MX
- Mexico
- Prior art keywords
- fabry disease
- galactosidase
- patient
- cell
- staining pattern
- 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
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61P17/00—Drugs for dermatological disorders
-
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
Abstract
La presente invención proporciona un método para tratar a un paciente con la enfermedad de Fabry, que comprende determinar si se produce una mejora en un marcador indirecto asociado con la enfermedad de Fabry después de la administración de una chaperona farmacológica específica de a-galactosidasa A. El método incluye administrar al individuo una cantidad efectiva de 1-desoxigalactonojirimicina, donde la 1-desoxigalactonojirimicina se une a alfa-galactosidasa A en una cantidad efectiva como para aumentar la actividad de la alfa-galactosidasa A. La presente invención proporciona también un método para supervisar y aumentar la respuesta terapéutica de un paciente con la enfermedad de Fabry después de la administración de una chaperona farmacológica esp0ecífica de a-palactosidasa A mediante la evaluación del efecto sobre el patrón de tinción citoplasmático de una célula del paciente, donde la detección de un patrón de tinción en la célula que es similar al patrón de tinción de una célula de un individuo sano indica que el in dividuo con la enfermedad de Fabry es un respondedor.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US90918507P | 2007-03-30 | 2007-03-30 | |
| PCT/US2008/058668 WO2008121826A2 (en) | 2007-03-30 | 2008-03-28 | Method for the treatment of fabry disease using pharmacological chaperones |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2009010557A true MX2009010557A (es) | 2009-11-19 |
Family
ID=39808872
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2009010557A MX2009010557A (es) | 2007-03-30 | 2008-03-28 | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20100113517A1 (es) |
| EP (1) | EP2142197A4 (es) |
| JP (1) | JP2010523578A (es) |
| AU (1) | AU2008232614A1 (es) |
| CA (1) | CA2682441A1 (es) |
| MX (1) | MX2009010557A (es) |
| WO (1) | WO2008121826A2 (es) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2533050T6 (da) | 2006-05-16 | 2015-09-21 | Amicus Therapeutics Inc | Behandlingsmuligheder ved Fabrys sygdom |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| EP2150254A4 (en) * | 2007-04-26 | 2010-11-10 | Amicus Therapeutics Inc | DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES |
| US8815580B2 (en) * | 2008-08-08 | 2014-08-26 | Vib Vzw | Cells producing glycoproteins having altered glycosylation patterns and method and use thereof |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| WO2010095940A2 (en) * | 2009-02-20 | 2010-08-26 | To-Bbb Holding B.V. | Glutathione-based drug delivery system |
| US20120195876A1 (en) * | 2009-08-14 | 2012-08-02 | Jochen Reiser | Novel role of alpha-galactosidase activity as a biomarker in kidney disease |
| TW201117810A (en) * | 2009-10-01 | 2011-06-01 | Baylor Res Inst | Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease |
| ES2768995T3 (es) * | 2009-11-17 | 2020-06-24 | Baylor Res Institute | 1-desoxigalactonojirimicina para el uso en el tratamiento de una enfermedad cardíaca |
| AU2010324810B2 (en) * | 2009-11-27 | 2016-05-12 | Genzyme Corporation | An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase |
| US20120178105A1 (en) * | 2011-01-10 | 2012-07-12 | Genzyme Corporation | Detection of globotriaosylceramide (glc) in human urine samples using an antibody sandwich |
| SG193379A1 (en) * | 2011-03-11 | 2013-10-30 | Amicus Therapeutics Inc | Dosing regimens for the treatment of fabry disease |
| US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| US9694056B2 (en) | 2012-07-17 | 2017-07-04 | Amicus Therapeutics, Inc. | α-galactosidase A and 1-deoxygalactonojirimycin co-formulation |
| WO2014120900A1 (en) * | 2013-01-31 | 2014-08-07 | Icahn School Of Medicine At Mount Sinai | Enhanced therapeutic regimens for treating fabry disease |
| SG10201804952QA (en) * | 2013-12-11 | 2018-07-30 | Genzyme Corp | Glucosylceramide synthase inhibitors |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| EP3432882B1 (en) * | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
| US20190183869A1 (en) | 2016-07-19 | 2019-06-20 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| EP4324522A2 (en) | 2017-05-30 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| KR20200044908A (ko) | 2017-08-28 | 2020-04-29 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
| PL3749308T3 (pl) * | 2018-02-06 | 2024-03-25 | Amicus Therapeutics, Inc. | Leczenie pacjentów z klasyczną chorobą fabry’ego migalastatem |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| US20210315875A1 (en) | 2018-08-20 | 2021-10-14 | Amicus Therapeutics, Inc. | Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
| 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 |
| CA3141226A1 (en) | 2019-06-11 | 2020-12-17 | Franklin Johnson | 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 |
| CA3170718A1 (en) | 2020-03-06 | 2021-09-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| AU2021311131A1 (en) | 2020-07-24 | 2023-03-23 | Genzyme Corporation | Pharmaceutical compositions comprising venglustat |
| WO2022132992A1 (en) | 2020-12-16 | 2022-06-23 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade 1-deoxygalactonojirimycin compounds |
| CA3225511A1 (en) | 2021-07-12 | 2023-01-19 | Franklin Johnson | Methods of treating fabry disease in pediatric patients |
Family Cites Families (1)
| 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 |
-
2008
- 2008-03-28 CA CA002682441A patent/CA2682441A1/en not_active Abandoned
- 2008-03-28 WO PCT/US2008/058668 patent/WO2008121826A2/en active Application Filing
- 2008-03-28 JP JP2010502218A patent/JP2010523578A/ja not_active Withdrawn
- 2008-03-28 MX MX2009010557A patent/MX2009010557A/es not_active Application Discontinuation
- 2008-03-28 EP EP08732987A patent/EP2142197A4/en not_active Withdrawn
- 2008-03-28 US US12/594,124 patent/US20100113517A1/en not_active Abandoned
- 2008-03-28 AU AU2008232614A patent/AU2008232614A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| AU2008232614A1 (en) | 2008-10-09 |
| EP2142197A4 (en) | 2010-11-10 |
| JP2010523578A (ja) | 2010-07-15 |
| WO2008121826A3 (en) | 2008-11-27 |
| WO2008121826A2 (en) | 2008-10-09 |
| CA2682441A1 (en) | 2008-10-09 |
| US20100113517A1 (en) | 2010-05-06 |
| EP2142197A2 (en) | 2010-01-13 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |