IL201733A0 - Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones - Google Patents
Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperonesInfo
- Publication number
- IL201733A0 IL201733A0 IL201733A IL20173309A IL201733A0 IL 201733 A0 IL201733 A0 IL 201733A0 IL 201733 A IL201733 A IL 201733A IL 20173309 A IL20173309 A IL 20173309A IL 201733 A0 IL201733 A0 IL 201733A0
- Authority
- IL
- Israel
- Prior art keywords
- treatment
- lysosomal storage
- dosing regimens
- storage diseases
- pharmacological chaperones
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 102000005431 Molecular Chaperones Human genes 0.000 title 1
- 108010006519 Molecular Chaperones Proteins 0.000 title 1
- 230000000144 pharmacologic effect Effects 0.000 title 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Psychology (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 |
|---|---|
| IL201733A0 true IL201733A0 (en) | 2010-06-16 |
Family
ID=39926311
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL201733A IL201733A0 (en) | 2007-04-26 | 2009-10-25 | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9056101B2 (enExample) |
| EP (1) | EP2150254A4 (enExample) |
| JP (1) | JP2010525084A (enExample) |
| AU (1) | AU2008245578A1 (enExample) |
| CA (1) | CA2685332A1 (enExample) |
| IL (1) | IL201733A0 (enExample) |
| MX (1) | MX2009011473A (enExample) |
| WO (1) | WO2008134628A2 (enExample) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI1589993T1 (sl) | 2003-01-31 | 2015-05-29 | Mount Sinai School Of Medicine Of New York University | Kombinirana terapija za zdravljenje motenj pomanjkanja proteina |
| HUE071237T2 (hu) | 2006-05-16 | 2025-08-28 | Amicus Therapeutics Inc | Fabry-kór kezelési lehetõségei |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| SI2946785T1 (sl) | 2008-02-12 | 2019-02-28 | Amicus Therapeutics, Inc. | Postopek napovedovanja odziva na farmakološko zdravljenje bolezni s šaperoni |
| BRPI0914684C1 (pt) | 2008-06-26 | 2021-05-25 | Orphazyme As | uso de hsp70 ou um fragmento funcional ou variante do mesmo, e, hsp70 ou um fragmento funcional ou variante do mesmo |
| 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 |
| NZ595629A (en) * | 2009-04-09 | 2013-04-26 | Amicus Therapeutics Inc | Methods for preventing and/or treating lysosomal storage disorders |
| GB0906159D0 (en) * | 2009-04-09 | 2009-05-20 | Summit Corp Plc | Drug combination for the treatment of proteostatic diseases |
| DK2490712T3 (en) | 2009-10-19 | 2015-10-05 | Amicus Therapeutics Inc | PROCESS FOR THE TREATMENT OF ALZHEIMER'S DISEASE BY USING Pharmacological chaperones TO INCREASE ACTIVITY OF GANGLIOSIDASER |
| KR101848938B1 (ko) | 2009-10-19 | 2018-04-13 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 리소좀 축적 질환을 예방 및/또는 치료하는 신규 조성물 |
| MX368459B (es) | 2009-10-19 | 2019-10-03 | Amicus Therapeutics Inc | Nuevas composiciones para prevenir y/o tratar trastornos degenerativos del sistema nervioso central. |
| DK2493487T3 (en) * | 2009-10-27 | 2016-12-05 | Erytech Pharma | Composition for induction of specific immunological tolerance |
| US9066939B2 (en) * | 2009-11-17 | 2015-06-30 | Baylor Research Institute | Urinary triaosylceramide (GB3) as a marker of cardiac disease |
| BR112012012947B8 (pt) * | 2009-11-27 | 2021-05-25 | Genzyme Corp | sal de hemitartarato e composição farmacêutica compreendendo o mesmo |
| CN103314105B (zh) | 2010-11-08 | 2015-11-25 | 阿米库斯治疗学公司 | 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白 |
| RU2013125923A (ru) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| SG10201604757RA (en) * | 2011-03-11 | 2016-08-30 | Amicus Therapeutics Inc | Dosing regimens for the treatment of fabry disease |
| EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
| MX349992B (es) | 2012-03-07 | 2017-08-22 | Amicus Therapeutics Inc | Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe. |
| AU2013240486B2 (en) | 2012-03-27 | 2017-10-26 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
| PT2844279T (pt) * | 2012-05-03 | 2021-03-11 | Amicus Therapeutics Inc | Esquema posológico para o tratamento da doença de pompe |
| CA2917995C (en) | 2012-07-17 | 2021-01-26 | Amicus Therapeutics, Inc. | Alpha-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 |
| EP3922242A1 (en) | 2014-09-15 | 2021-12-15 | Orphazyme A/S | Arimoclomol formulation |
| HRP20250058T1 (hr) | 2014-09-30 | 2025-03-28 | Amicus Therapeutics, Inc. | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
| 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 |
| WO2017041051A1 (en) | 2015-09-04 | 2017-03-09 | Sqz Biotechnologies Company | Intracellular delivery of biomolecules to cells comprising a cell wall |
| KR20250007693A (ko) | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| 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 |
| JP7510743B2 (ja) | 2016-03-30 | 2024-07-04 | アミカス セラピューティックス インコーポレイテッド | 組換え酸性α－グルコシダーゼを含む製剤 |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| BR112018070653A2 (pt) | 2016-04-29 | 2019-02-05 | Orphazyme As | ingrediente farmacêutico ativo, e, composição |
| KR102430856B1 (ko) | 2016-05-03 | 2022-08-08 | 에스큐지 바이오테크놀로지스 컴퍼니 | 관용을 유도하는 생체분자의 세포내 전달 |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| AU2018258348B2 (en) | 2017-04-25 | 2022-07-07 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders |
| HRP20230576T1 (hr) | 2017-05-15 | 2023-09-01 | Amicus Therapeutics, Inc. | Rekombinantna ljudska kisela alfa-glukozidaza |
| LT4062916T (lt) * | 2017-05-30 | 2025-01-10 | Amicus Therapeutics, Inc. | Tam tikras mutacijas turinčių fabri pacientų gydymas |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of 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 |
| WO2020123511A2 (en) * | 2018-12-10 | 2020-06-18 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| EA202290024A1 (ru) * | 2019-06-11 | 2022-03-14 | Амикус Терапьютикс, Инк. | Способы лечения болезни фабри у пациентов с почечной недостаточностью |
| 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 |
| AU2021380947C1 (en) | 2020-11-19 | 2025-02-20 | 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 |
| SI1589993T1 (sl) * | 2003-01-31 | 2015-05-29 | 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 |
| KR20080025373A (ko) | 2005-05-17 | 2008-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 1-데옥시노지리마이신 및 유도체를 이용한 폼페병의 치료방법 |
| US7964617B2 (en) * | 2005-06-08 | 2011-06-21 | Amicus Therapeutics, Inc. | Methods for treating parkinsons disease and parkinsonism |
| 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 |
| HUE071237T2 (hu) * | 2006-05-16 | 2025-08-28 | Amicus Therapeutics Inc | Fabry-kór kezelési lehetõségei |
| WO2008121826A2 (en) | 2007-03-30 | 2008-10-09 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease using pharmacological chaperones |
-
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 WO PCT/US2008/061764 patent/WO2008134628A2/en not_active Ceased
- 2008-04-28 CA CA002685332A patent/CA2685332A1/en not_active Abandoned
- 2008-04-28 AU AU2008245578A patent/AU2008245578A1/en not_active Abandoned
- 2008-04-28 JP JP2010506557A patent/JP2010525084A/ja active Pending
- 2008-04-28 EP EP08747020A patent/EP2150254A4/en not_active Withdrawn
-
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 |
|---|---|
| AU2008245578A1 (en) | 2008-11-06 |
| US20100266571A1 (en) | 2010-10-21 |
| CA2685332A1 (en) | 2008-11-06 |
| WO2008134628A2 (en) | 2008-11-06 |
| US9056101B2 (en) | 2015-06-16 |
| MX2009011473A (es) | 2010-01-18 |
| EP2150254A2 (en) | 2010-02-10 |
| WO2008134628A3 (en) | 2010-01-14 |
| JP2010525084A (ja) | 2010-07-22 |
| EP2150254A4 (en) | 2010-11-10 |
| US20150352093A1 (en) | 2015-12-10 |
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