PT2332567E - Terapia combinada para o tratamento de distúrbios de deficiência proteica - Google Patents
Terapia combinada para o tratamento de distúrbios de deficiência proteica Download PDFInfo
- Publication number
- PT2332567E PT2332567E PT100115989T PT04011598T PT2332567E PT 2332567 E PT2332567 E PT 2332567E PT 100115989 T PT100115989 T PT 100115989T PT 04011598 T PT04011598 T PT 04011598T PT 2332567 E PT2332567 E PT 2332567E
- Authority
- PT
- Portugal
- Prior art keywords
- combination therapy
- protein deficiency
- deficiency disorders
- treating protein
- treating
- Prior art date
Links
Classifications
-
- 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
- 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
- A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/02—Nutrients, e.g. vitamins, minerals
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- 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
- A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Emergency Medicine (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Nutrition Science (AREA)
- Endocrinology (AREA)
- Vascular Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US44413603P | 2003-01-31 | 2003-01-31 |
Publications (1)
Publication Number | Publication Date |
---|---|
PT2332567E true PT2332567E (pt) | 2015-09-23 |
Family
ID=32850826
Family Applications (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT110064953T PT2444102E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT04707422T PT1589993E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT141971978T PT2857036T (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT100115989T PT2332567E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT110065042T PT2441467E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica |
Family Applications Before (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT110064953T PT2444102E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT04707422T PT1589993E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
PT141971978T PT2857036T (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT110065042T PT2441467E (pt) | 2003-01-31 | 2004-02-02 | Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica |
Country Status (16)
Country | Link |
---|---|
US (4) | US20040180419A1 (pt) |
EP (6) | EP2857036B1 (pt) |
JP (5) | JP5589165B2 (pt) |
CN (4) | CN102580067A (pt) |
BR (1) | BRPI0407001A (pt) |
CA (3) | CA2814767C (pt) |
CY (5) | CY1116215T1 (pt) |
DK (5) | DK2444102T3 (pt) |
ES (6) | ES2541952T3 (pt) |
HK (4) | HK1158955A1 (pt) |
HU (2) | HUE027113T2 (pt) |
IL (6) | IL169562A (pt) |
MX (2) | MX344587B (pt) |
PT (5) | PT2444102E (pt) |
SI (5) | SI1589993T1 (pt) |
WO (1) | WO2004069190A2 (pt) |
Families Citing this family (54)
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US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US20020095135A1 (en) * | 2000-06-19 | 2002-07-18 | David Meeker | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
PT2444102E (pt) * | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
FR2861991B1 (fr) | 2003-11-07 | 2008-01-18 | Centre Nat Rech Scient | Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose |
US20050256168A1 (en) * | 2004-04-28 | 2005-11-17 | Block Timothy M | Compositions for oral administration for the treatment of interferon-responsive disorders |
EP3441090A1 (en) | 2005-05-17 | 2019-02-13 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
JP2009509913A (ja) | 2005-06-08 | 2009-03-12 | アミカス セラピューティックス インコーポレイテッド | リソソーム酵素をコードする遺伝子の変異に関連するcns障害の治療 |
JP5364382B2 (ja) * | 2006-02-07 | 2013-12-11 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 遊離チオール部分を有するタンパク質の安定化された組成物 |
PL2787345T3 (pl) | 2006-05-16 | 2016-08-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry’ego |
ATE555788T1 (de) * | 2006-06-23 | 2012-05-15 | Amicus Therapeutics Inc | Verfahren zur behandlung neurologischer erkrankungen mittels verstärkung der beta - glucocerebrosidaseaktivität |
AU2007277032A1 (en) * | 2006-07-27 | 2008-01-31 | University Of Florida Research Foundation, Inc. | Opsin stabilizing compounds and methods of use |
WO2008013984A2 (en) * | 2006-07-27 | 2008-01-31 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating or preventing ophthalmic disease |
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JP2010525084A (ja) * | 2007-04-26 | 2010-07-22 | アミカス セラピューティックス インコーポレイテッド | 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 |
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US9206457B2 (en) * | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
CA2763464C (en) * | 2009-05-26 | 2018-11-06 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
CA2765086C (en) | 2009-06-12 | 2015-12-15 | United Therapeutics Corporation | Iminosugars and methods of treating bunyaviral and togaviral diseases |
IN2012DN00352A (pt) * | 2009-06-16 | 2015-08-21 | Bikam Pharmaceuticals Inc | |
EP2459212B1 (en) | 2009-07-28 | 2018-03-14 | Shire Human Genetic Therapies, Inc. | Compositions and methods for treating gaucher disease |
CA2772875A1 (en) * | 2009-09-04 | 2011-03-10 | United Therapeutics Corporation | Methods of treating orthomyxoviral infections |
ES2485623T3 (es) * | 2009-09-04 | 2014-08-13 | United Therapeutics Corporation | Procedimiento para el tratamiento de infecciones por poxvirus |
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HRP20211520T1 (hr) | 2010-06-25 | 2021-12-24 | Shire Human Genetic Therapies, Inc. | Postupci i pripravci za dostavu iduronat-2-sulfataze u središnji živčani sustav |
CN103179980B (zh) * | 2010-06-25 | 2016-09-28 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物 |
US20140219986A1 (en) * | 2011-03-11 | 2014-08-07 | Amicus Therapeutics ,Inc. | Dosing regimens for the treatment of fabry disease |
US20150258081A1 (en) * | 2011-12-22 | 2015-09-17 | Centogene Ip Gmbh | Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol |
CN104519905A (zh) | 2012-03-02 | 2015-04-15 | 夏尔人类遗传性治疗公司 | 用于治疗iii型戈谢病的组合物和方法 |
KR20200032244A (ko) | 2012-03-07 | 2020-03-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
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PT3957320T (pt) | 2015-12-30 | 2023-11-23 | Amicus Therapeutics Inc | Alfa-glicosidase ácida aumentada para o tratamento da doença de pompe |
KR20230041833A (ko) | 2015-12-30 | 2023-03-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
KR20220145918A (ko) | 2016-03-30 | 2022-10-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
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US11590131B2 (en) | 2017-07-28 | 2023-02-28 | Applied Therapeutics, Inc. | Compositions and methods for treating galactosemia |
US20200316178A1 (en) * | 2017-10-26 | 2020-10-08 | Shire Human Genetic Therapies, Inc. | Formulations comprising glucocerebrosidase and isofagomine |
WO2020046132A1 (en) * | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
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SG11202111822QA (en) * | 2019-05-07 | 2021-11-29 | Univ Miami | Treatment and detection of inherited neuropathies and associated disorders |
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PT2444102E (pt) * | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
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EP3441090A1 (en) * | 2005-05-17 | 2019-02-13 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
JP2010525084A (ja) | 2007-04-26 | 2010-07-22 | アミカス セラピューティックス インコーポレイテッド | 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 |
-
2004
- 2004-02-02 PT PT110064953T patent/PT2444102E/pt unknown
- 2004-02-02 WO PCT/US2004/002973 patent/WO2004069190A2/en active Application Filing
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