BR0309665A - Tratamento de deficiência de alfa-galactosidase a - Google Patents
Tratamento de deficiência de alfa-galactosidase aInfo
- Publication number
- BR0309665A BR0309665A BR0309665-3A BR0309665A BR0309665A BR 0309665 A BR0309665 A BR 0309665A BR 0309665 A BR0309665 A BR 0309665A BR 0309665 A BR0309665 A BR 0309665A
- Authority
- BR
- Brazil
- Prior art keywords
- treatment
- alpha
- galactosidase
- deficiency
- galactosidase deficiency
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title 1
- 230000007812 deficiency Effects 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 238000004458 analytical method Methods 0.000 abstract 1
- 239000002552 dosage form Substances 0.000 abstract 1
Classifications
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- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/12—Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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)
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P25/04—Centrally acting analgesics, e.g. opioids
-
- 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
- A61P3/06—Antihyperlipidemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/02—Antidotes
-
- 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
-
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/54—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving glucose or galactose
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2400/00—Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
- G01N2400/10—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Molecular Biology (AREA)
- Diabetes (AREA)
- Urology & Nephrology (AREA)
- Analytical Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Obesity (AREA)
- Biophysics (AREA)
- Emergency Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Cardiology (AREA)
- Toxicology (AREA)
Abstract
"TRATAMENTO DE DEFICIêNCIA DE ALFAGALACTOSIDASE A". A invenção provê métodos de tratamento de deficiência de <244>-galactosidase A. Formas de dosagem, métodos de administração e métodos de análise de <244>-galactosidase A humana são também incluídos.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US37558402P | 2002-04-25 | 2002-04-25 | |
US60/375,584 | 2002-04-25 | ||
PCT/US2003/013063 WO2003090695A2 (en) | 2002-04-25 | 2003-04-25 | TREATMENT OF α-GALACTOSIDASE A DEFICIENCY |
Publications (3)
Publication Number | Publication Date |
---|---|
BR0309665A true BR0309665A (pt) | 2005-08-16 |
BRPI0309665B1 BRPI0309665B1 (pt) | 2019-07-16 |
BRPI0309665B8 BRPI0309665B8 (pt) | 2021-05-25 |
Family
ID=29270665
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BRPI0309665A BRPI0309665B8 (pt) | 2002-04-25 | 2003-04-25 | método para analisar uma preparação de alfagalactosidase a |
BR122017016940A BR122017016940B8 (pt) | 2002-04-25 | 2003-04-25 | método para preparação de uma composição farmacêutica compreendendo alfa-galactosidase a |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR122017016940A BR122017016940B8 (pt) | 2002-04-25 | 2003-04-25 | método para preparação de uma composição farmacêutica compreendendo alfa-galactosidase a |
Country Status (13)
Country | Link |
---|---|
US (6) | US20040071686A1 (pt) |
EP (2) | EP2361631A1 (pt) |
JP (5) | JP5570677B2 (pt) |
AT (1) | ATE514430T1 (pt) |
AU (2) | AU2003228722B2 (pt) |
BR (2) | BRPI0309665B8 (pt) |
CA (1) | CA2483270C (pt) |
DK (1) | DK1503788T3 (pt) |
ES (1) | ES2367257T3 (pt) |
HK (1) | HK1073608A1 (pt) |
IL (4) | IL164757A0 (pt) |
NZ (1) | NZ536361A (pt) |
WO (1) | WO2003090695A2 (pt) |
Families Citing this family (38)
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CA2483270C (en) * | 2002-04-25 | 2015-03-31 | Transkaryotic Therapies, Inc. | Treatment of alpha-galactosidase a deficiency |
US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
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AU2005295467B2 (en) * | 2004-10-15 | 2011-07-07 | Velico Medical, Inc. | Compositions and methods for prolonging survival of platelets |
WO2007097934A2 (en) * | 2006-02-17 | 2007-08-30 | Elusys Therapeutics, Inc. | Methods and compositions for using erythrocytes as carriers for delivery of drugs |
US20100144008A1 (en) * | 2006-12-21 | 2010-06-10 | Academisch Ziekenhuis Bij De Universiteit Van Amsterdam | Treatment of fabry disease |
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US20100291060A1 (en) * | 2007-08-29 | 2010-11-18 | Shire Human Genetic Therapies, Inc | Subcutaneous administration of alpha-galactosidase a |
WO2011061736A1 (en) * | 2009-11-17 | 2011-05-26 | Protalix Ltd. | Alkaline alpha galactosidase for the treatment of fabry disease |
US9194011B2 (en) | 2009-11-17 | 2015-11-24 | Protalix Ltd. | Stabilized alpha-galactosidase and uses thereof |
RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
KR102211960B1 (ko) * | 2010-06-25 | 2021-02-05 | 샤이어 휴먼 지네틱 테라피즈 인크. | 치료제들의 cns 전달 |
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KR102094094B1 (ko) | 2010-06-25 | 2020-03-27 | 샤이어 휴먼 지네틱 테라피즈 인크. | 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 |
AU2012204526B2 (en) | 2011-01-03 | 2016-05-19 | California Institute Of Technology | High density epidural stimulation for facilitation of locomotion, posture, voluntary movement, and recovery of autonomic, sexual, vasomotor, and cognitive function after neurological injury |
BR112013018516B1 (pt) | 2011-01-20 | 2023-11-07 | Protalix Ltd | Construto de expressão de ácido nucleico, método de produção de uma proteína alfa-galactosidase humana recombinante, proteína alfagalactosidase humana, composição farmacêutica, e, uso de uma proteína alfagalactosidase humana ou de uma composição |
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WO2014014938A1 (en) * | 2012-07-17 | 2014-01-23 | 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 |
WO2015048563A2 (en) | 2013-09-27 | 2015-04-02 | The Regents Of The University Of California | Engaging the cervical spinal cord circuitry to re-enable volitional control of hand function in tetraplegic subjects |
WO2015066357A1 (en) * | 2013-10-31 | 2015-05-07 | Amgen Inc. | Use of monensin to regulate glycosylation of recombinant proteins |
BR112017013300A2 (pt) * | 2014-12-22 | 2018-02-27 | Codexis Inc | alfa galactosidase a recombinante e/ou fragmento de alfa galactosidase a recombinante biologicamente ativo, composição, sequência de polinucleotídeos recombinantes, vetor de expressão, célula hospedeira, métodos para produzir uma variante de alfa galactosidase a e para tratar e/ou prevenir os sintomas da doença de fabry, composição farmacêutica, e, uso das composições. |
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GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
JP6996676B2 (ja) * | 2016-03-30 | 2022-02-04 | 国立研究開発法人農業・食品産業技術総合研究機構 | マンノース-6-リン酸基含有糖蛋白質の製造方法、及び蛍光基結合型マンノース-6-リン酸基含有糖蛋白質の細胞内分布を検出する方法 |
KR20240042110A (ko) | 2017-01-05 | 2024-04-01 | 프로탈릭스 리미티드 | 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법 |
US11357834B2 (en) | 2017-01-10 | 2022-06-14 | Amicus Therapeutics, Inc. | Recombinant α-galactosidase A for treatment of Fabry disease |
EP3421081B1 (en) | 2017-06-30 | 2020-04-15 | GTX medical B.V. | A system for neuromodulation |
JP7252211B2 (ja) * | 2017-09-08 | 2023-04-04 | ザ ヌムール ファウンデーション | リソソーム蓄積症を治療するための薬剤、デバイス、および血液循環システム、ならびにリソソーム蓄積症を治療するための方法 |
WO2019110400A1 (en) | 2017-12-05 | 2019-06-13 | Ecole Polytechnique Federale De Lausanne (Epfl) | A system for planning and/or providing neuromodulation |
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BR112022016990A2 (pt) * | 2020-02-28 | 2022-10-25 | Codexis Inc | Alfa-galactosidases a recombinantes e/ou fragmento de alfa-galactosidase a recombinante biologicamente ativo, composição, sequência de polinucleotídeo recombinante, vetor de expressão, célula hospedeira, métodos para produzir uma variante de alfa-galactosidase a e para tratar e/ou prevenir os sintomas de doença de fabry em um indivíduo, composição farmacêutica para o tratamento de doença de fabry, composição farmacêutica, e, uso das composições |
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