MX2009011473A - Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. - Google Patents
Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.Info
- Publication number
- MX2009011473A MX2009011473A MX2009011473A MX2009011473A MX2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A MX 2009011473 A MX2009011473 A MX 2009011473A
- Authority
- MX
- Mexico
- Prior art keywords
- dosing regimens
- lysosomal storage
- treatment
- pharmacological chaperones
- storage diseases
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- 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
Abstract
La presente invención proporciona regímenes de dosificación para administrar chaperonas farmacológicas a un sujeto que lo necesite. Los regímenes de dosificación se pueden utilizar para tratar trastornos causados por un mal plegamiento de proteínas inadecuado, tales como trastornos por almacenamiento lisosomal.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US91428807P | 2007-04-26 | 2007-04-26 | |
US1474407P | 2007-12-18 | 2007-12-18 | |
US2810508P | 2008-02-12 | 2008-02-12 | |
PCT/US2008/061764 WO2008134628A2 (en) | 2007-04-26 | 2008-04-28 | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2009011473A true MX2009011473A (es) | 2010-01-18 |
Family
ID=39926311
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2009011473A MX2009011473A (es) | 2007-04-26 | 2008-04-28 | Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. |
Country Status (8)
Country | Link |
---|---|
US (2) | US9056101B2 (es) |
EP (1) | EP2150254A4 (es) |
JP (1) | JP2010525084A (es) |
AU (1) | AU2008245578A1 (es) |
CA (1) | CA2685332A1 (es) |
IL (1) | IL201733A0 (es) |
MX (1) | MX2009011473A (es) |
WO (1) | WO2008134628A2 (es) |
Families Citing this family (45)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2332567B1 (en) | 2003-01-31 | 2015-04-08 | The Mount Sinai School of Medicine of New York University | Combination therapy for treating protein deficiency disorders |
ES2464527T7 (es) | 2006-05-16 | 2015-09-14 | Amicus Therapeutics, Inc. | Opciones de tratamiento para la enfermedad de Fabry |
US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
PL2946785T3 (pl) | 2008-02-12 | 2019-04-30 | Amicus Therapeutics Inc | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
BR122019024895B8 (pt) | 2008-06-26 | 2021-07-27 | Orphazyme As | uso de um agente bioativo capaz de aumentar a concentração intracelular de hsp70 |
WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
US20100119502A1 (en) * | 2008-11-11 | 2010-05-13 | Amicus Therapeutics, Inc. | Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease |
BRPI1010517A2 (pt) * | 2009-04-09 | 2017-01-31 | Amicus Therapeutics Inc | métodos para prevenção e/ou tratamento de doenças de armazenamento lisossomal |
GB0906159D0 (en) * | 2009-04-09 | 2009-05-20 | Summit Corp Plc | Drug combination for the treatment of proteostatic diseases |
US9044437B2 (en) * | 2009-10-19 | 2015-06-02 | Amicus Therapeutics, Inc. | Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases |
MX368459B (es) | 2009-10-19 | 2019-10-03 | Amicus Therapeutics Inc | Nuevas composiciones para prevenir y/o tratar trastornos degenerativos del sistema nervioso central. |
CN102647905B (zh) | 2009-10-19 | 2015-09-09 | 阿米库斯治疗学公司 | 用于预防和/或治疗溶酶体贮积失调的新颖组合物 |
ES2600932T3 (es) * | 2009-10-27 | 2017-02-13 | Erytech Pharma | Composición para inducir una tolerancia inmunitaria específica |
EP2502068B1 (en) * | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
SI2796457T1 (sl) * | 2009-11-27 | 2016-10-28 | Genzyme Corporation | Genz 112638 za zdravljenje Gaucherjeve ali Fabryjeve bolezni v kombinirani terapiji |
CA2817011C (en) * | 2010-11-08 | 2019-04-02 | Callidus Biopharma, Inc. | Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity |
EP3626255A1 (en) | 2010-11-30 | 2020-03-25 | Orphazyme A/S | Methods for increasing intracellular activity of hsp70 |
ES2807502T3 (es) * | 2011-03-11 | 2021-02-23 | Amicus Therapeutics Inc | Regímenes de dosificación para el tratamiento de la enfermedad de Fabry |
EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
WO2013148103A1 (en) | 2012-03-27 | 2013-10-03 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
SI2844279T1 (sl) * | 2012-05-03 | 2021-04-30 | Amicus Therapeutics, Inc. | Dozirni režimi za zdravljenje Pompejeve bolezni |
US9694056B2 (en) | 2012-07-17 | 2017-07-04 | Amicus Therapeutics, Inc. | α-galactosidase A and 1-deoxygalactonojirimycin co-formulation |
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 |
US9675627B2 (en) | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
ES2881860T3 (es) | 2014-09-15 | 2021-11-30 | Orphazyme As | Formulación de arimoclomol |
RS65066B1 (sr) | 2014-09-30 | 2024-02-29 | Amicus Therapeutics Inc | Visoko potentna kisela alfa-glukozidaza sa pojačanim ugljenim hidratima |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
US10179128B2 (en) * | 2015-08-31 | 2019-01-15 | Amicus Therapeutics, Inc. | Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system |
EP3344575B1 (en) | 2015-09-04 | 2020-04-15 | SQZ Biotechnologies Company | Intracellular delivery of biomolecules to cells comprising a cell wall |
WO2017117407A1 (en) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
JP6438421B2 (ja) * | 2016-02-17 | 2018-12-12 | ファナック株式会社 | 電動機のステータ |
US10227577B2 (en) | 2016-03-30 | 2019-03-12 | Amicus Therapeutics, Inc. | Method for selection of high M6P recombinant proteins |
KR20240001291A (ko) | 2016-03-30 | 2024-01-03 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
WO2017178029A1 (en) | 2016-04-13 | 2017-10-19 | Orphazyme Aps | Heat shock proteins and cholesterol homeostasis |
LT3448382T (lt) | 2016-04-29 | 2021-04-12 | Orphazyme A/S | Arimoklomolis, skirtas su gliukocerebrozidaze susijusiems sutrikimams gydyti |
NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
EP3615509A1 (en) | 2017-04-25 | 2020-03-04 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders |
CN117357524A (zh) * | 2017-05-30 | 2024-01-09 | 阿米库斯治疗学公司 | 治疗具有肾损害的法布里患者的方法 |
CA3065298A1 (en) | 2017-05-30 | 2018-12-06 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
JP2022536687A (ja) * | 2019-06-11 | 2022-08-18 | アミカス セラピューティックス インコーポレイテッド | 腎機能障害を有する患者のファブリー病を治療する方法 |
MX2022001623A (es) | 2019-08-07 | 2022-03-02 | Amicus Therapeutics Inc | Metodos para tratar la enfermedad de fabry en pacientes que tienen una mutacion en el gen gla. |
AU2021380947B2 (en) | 2020-11-19 | 2023-12-14 | Zevra Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
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 (9)
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 |
EP2332567B1 (en) * | 2003-01-31 | 2015-04-08 | The 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 |
EP2932982B1 (en) * | 2005-05-17 | 2018-10-03 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
EP1888068B1 (en) * | 2005-06-08 | 2014-05-21 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
US7955262B2 (en) | 2005-07-26 | 2011-06-07 | Syneron Medical Ltd. | Method and apparatus for treatment of skin using RF and ultrasound energies |
KR20080033463A (ko) * | 2005-07-27 | 2008-04-16 | 유니버시티 오브 플로리다 리서치 파운데이션, 아이엔씨. | 단백질 미스폴딩을 보정하는 작은 화합물 및 그의 용도 |
ES2464527T7 (es) * | 2006-05-16 | 2015-09-14 | Amicus Therapeutics, Inc. | Opciones de tratamiento para la enfermedad de Fabry |
EP2142197A4 (en) | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS |
-
2008
- 2008-04-28 US US12/597,238 patent/US9056101B2/en active Active
- 2008-04-28 MX MX2009011473A patent/MX2009011473A/es not_active Application Discontinuation
- 2008-04-28 AU AU2008245578A patent/AU2008245578A1/en not_active Abandoned
- 2008-04-28 WO PCT/US2008/061764 patent/WO2008134628A2/en active Application Filing
- 2008-04-28 JP JP2010506557A patent/JP2010525084A/ja active Pending
- 2008-04-28 EP EP08747020A patent/EP2150254A4/en not_active Withdrawn
- 2008-04-28 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
-
2009
- 2009-10-25 IL IL201733A patent/IL201733A0/en unknown
-
2015
- 2015-05-15 US US14/713,821 patent/US20150352093A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
US9056101B2 (en) | 2015-06-16 |
AU2008245578A1 (en) | 2008-11-06 |
EP2150254A4 (en) | 2010-11-10 |
JP2010525084A (ja) | 2010-07-22 |
WO2008134628A3 (en) | 2010-01-14 |
IL201733A0 (en) | 2010-06-16 |
WO2008134628A2 (en) | 2008-11-06 |
US20150352093A1 (en) | 2015-12-10 |
CA2685332A1 (en) | 2008-11-06 |
US20100266571A1 (en) | 2010-10-21 |
EP2150254A2 (en) | 2010-02-10 |
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Legal Events
Date | Code | Title | Description |
---|---|---|---|
FA | Abandonment or withdrawal |