CY1121113T1 - Θεραπεια της νοσου ρομρε - Google Patents
Θεραπεια της νοσου ρομρεInfo
- Publication number
- CY1121113T1 CY1121113T1 CY20181100718T CY181100718T CY1121113T1 CY 1121113 T1 CY1121113 T1 CY 1121113T1 CY 20181100718 T CY20181100718 T CY 20181100718T CY 181100718 T CY181100718 T CY 181100718T CY 1121113 T1 CY1121113 T1 CY 1121113T1
- Authority
- CY
- Cyprus
- Prior art keywords
- romre
- disease treatment
- patient
- body weight
- provides methods
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 abstract 2
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 abstract 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 abstract 1
- 108010028144 alpha-Glucosidases Proteins 0.000 abstract 1
- 230000037396 body weight Effects 0.000 abstract 1
- 201000004502 glycogen storage disease II Diseases 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 238000011269 treatment regimen Methods 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
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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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0276—Knock-out vertebrates
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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- 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
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- 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
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
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- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/107—Rabbit
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/01—Animal expressing industrially exogenous proteins
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Environmental Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Cardiology (AREA)
- Dermatology (AREA)
- Endocrinology (AREA)
Abstract
Η εφεύρεση παρέχει μεθόδους θεραπείας της νόσου Pompe χρησιμοποιώντας ανθρώπινη άλφα γλυκοσιδάση. Ένα προτιμώμενο θεραπευτικό σχήμα περιλαμβάνει χορήγηση μεγαλύτερη από 10mg/kg σωματικού βάρους την εβδομάδα σε έναν ασθενή.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US11129198P | 1998-12-07 | 1998-12-07 | |
EP99965162A EP1137762B1 (en) | 1998-12-07 | 1999-12-06 | Treatment of pompe's disease |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1121113T1 true CY1121113T1 (el) | 2019-07-10 |
Family
ID=22337648
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20181100718T CY1121113T1 (el) | 1998-12-07 | 2018-07-10 | Θεραπεια της νοσου ρομρε |
CY2018020C CY2018020I2 (el) | 1998-12-07 | 2018-07-25 | Θεραπεια της νοσου ρομρε |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY2018020C CY2018020I2 (el) | 1998-12-07 | 2018-07-25 | Θεραπεια της νοσου ρομρε |
Country Status (16)
Country | Link |
---|---|
US (7) | US20030007963A1 (el) |
EP (2) | EP2020438B1 (el) |
JP (7) | JP4990434B2 (el) |
KR (1) | KR20010101131A (el) |
AT (1) | ATE410506T1 (el) |
AU (1) | AU3113700A (el) |
BE (1) | BE2018C038I2 (el) |
CA (1) | CA2353522A1 (el) |
CY (2) | CY1121113T1 (el) |
DE (2) | DE69939705D1 (el) |
DK (2) | DK2020438T3 (el) |
ES (2) | ES2312222T3 (el) |
LU (1) | LU91544I2 (el) |
NL (1) | NL300382I2 (el) |
PT (2) | PT1137762E (el) |
WO (1) | WO2000034451A1 (el) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU3113700A (en) * | 1998-12-07 | 2000-06-26 | Genzyme Corporation | Treatment of pompe's disease |
WO2001056596A1 (en) * | 2000-02-04 | 2001-08-09 | Children's Hospital Research Foundation | Use of lysosomal acid lipase for treating atherosclerosis and related diseases |
EP1301201B1 (en) * | 2000-07-18 | 2007-02-28 | Duke University | Treatment of glycogen storage disease type ii |
EP1782825B1 (en) * | 2000-07-18 | 2014-09-10 | Duke University | Treatment of glycogen storage diseases type II |
AU2016256663A1 (en) * | 2000-07-18 | 2016-11-24 | Duke University | Treatment of clycogen storage disease type II |
US7723296B2 (en) * | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7396811B2 (en) * | 2001-04-30 | 2008-07-08 | Zystor Therapeutics, Inc. | Subcellular targeting of therapeutic proteins |
US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
US7658916B2 (en) | 2002-04-05 | 2010-02-09 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density |
ES2371913T3 (es) * | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
CA2814767C (en) * | 2003-01-31 | 2016-03-15 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
ATE465250T1 (de) * | 2004-02-10 | 2010-05-15 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
KR100697270B1 (ko) * | 2004-12-10 | 2007-03-21 | 삼성전자주식회사 | 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법 |
CA2612538C (en) | 2005-05-17 | 2015-06-30 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
CA2669347A1 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Methods for treating pompe disease |
BR122017015900A2 (pt) | 2008-05-07 | 2019-09-10 | Biomarin Pharm Inc | ácido nucleico e células |
EP2318037B1 (en) | 2008-07-08 | 2015-01-28 | Duke University | Method of treating glycogen storage disease |
ES2569514T3 (es) | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
WO2011028941A2 (en) | 2009-09-04 | 2011-03-10 | The United States Of America, As Represented By The Secretary Department Of Health & Human Services | Disabling autophagy as a treatment for lysosomal storage diseases |
WO2011133960A2 (en) | 2010-04-23 | 2011-10-27 | Synageva Biopharma Corp | Lysosomal storage disease enzyme |
PE20180130A1 (es) | 2010-06-25 | 2018-01-18 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a |
KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
ME03647B (me) | 2010-06-25 | 2020-07-20 | Shire Human Genetic Therapies | Postupci i kompozicije za isporuku arilsulfataze a u cns |
KR20140005842A (ko) | 2010-06-25 | 2014-01-15 | 샤이어 휴먼 지네틱 테라피즈 인크. | 헤파란 n-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
WO2011163652A2 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
KR20240068752A (ko) | 2010-06-25 | 2024-05-17 | 샤이어 휴먼 지네틱 테라피즈 인크. | 치료제들의 cns 전달 |
NZ700824A (en) | 2010-09-09 | 2016-03-31 | Synageva Biopharma Corp | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
WO2012124520A1 (ja) * | 2011-03-16 | 2012-09-20 | 天野エンザイム株式会社 | 改変型α-グルコシダーゼ及びその用途 |
AU2012245280A1 (en) * | 2011-04-22 | 2013-11-07 | Genzyme Corporation | Modified acid alpha glucosidase with accelerated processing |
WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
WO2014130723A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of pompe disease |
WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
EA038986B1 (ru) | 2014-09-30 | 2021-11-18 | Амикус Терапьютикс, Инк. | Высокоактивная кислая альфа-глюкозидаза с повышенным содержанием углеводов |
MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
WO2017040647A1 (en) * | 2015-08-31 | 2017-03-09 | Duke University | Methods and compositions for the treatment of cytopplasmic glycogen storage disorders |
WO2017049157A1 (en) | 2015-09-18 | 2017-03-23 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
JP2019501178A (ja) | 2015-12-30 | 2019-01-17 | アミカス セラピューティックス インコーポレイテッド | ポンペ病の処置のための強化酸性アルファ−グルコシダーゼ |
KR102343162B1 (ko) * | 2016-03-30 | 2021-12-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
KR102617502B1 (ko) | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
US20220331408A1 (en) | 2019-07-09 | 2022-10-20 | Genethon | Treatment of glycogen storage disease (gsd) |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP0832981A1 (en) | 1987-02-17 | 1998-04-01 | Pharming B.V. | DNA sequences to target proteins to the mammary gland for efficient secretion |
WO1993025567A1 (en) | 1992-06-15 | 1993-12-23 | Gene Pharming Europe B.V. | Production of recombinant polypeptides by bovine species and transgenic methods |
CA2351200A1 (en) | 1989-12-01 | 1991-06-13 | Gene Pharming Europe Bv | Production of recombinant polypeptides by bovine species and transgenic methods |
US5612205A (en) | 1990-08-29 | 1997-03-18 | Genpharm International, Incorporated | Homologous recombination in mammalian cells |
US5356804A (en) | 1990-10-24 | 1994-10-18 | Mount Sinai School Of Medicine Of The City Of New York | Cloning and expression of biologically active human α-galactosidase A |
CA2123671C (en) * | 1993-05-17 | 1999-08-03 | Mitsuo Yamada | Oxazolidone ring-containing modified epoxy resins and cathodic electrodeposition paints containing same |
AT1470U1 (de) | 1995-08-01 | 1997-05-26 | Austria Card | Laminierte karte und verfahren zu ihrer herstellung |
US6118045A (en) * | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
AU3113700A (en) * | 1998-12-07 | 2000-06-26 | Genzyme Corporation | Treatment of pompe's disease |
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1999
- 1999-12-06 AU AU31137/00A patent/AU3113700A/en not_active Abandoned
- 1999-12-06 DE DE69939705T patent/DE69939705D1/de not_active Expired - Lifetime
- 1999-12-06 ES ES99965162T patent/ES2312222T3/es not_active Expired - Lifetime
- 1999-12-06 KR KR1020017007040A patent/KR20010101131A/ko not_active Application Discontinuation
- 1999-12-06 ES ES08164825.5T patent/ES2677343T3/es not_active Expired - Lifetime
- 1999-12-06 EP EP08164825.5A patent/EP2020438B1/en not_active Revoked
- 1999-12-06 DK DK08164825.5T patent/DK2020438T3/en active
- 1999-12-06 AT AT99965162T patent/ATE410506T1/de active
- 1999-12-06 DE DE122009000018C patent/DE122009000018I1/de active Pending
- 1999-12-06 DK DK99965162T patent/DK1137762T3/da active
- 1999-12-06 CA CA002353522A patent/CA2353522A1/en not_active Abandoned
- 1999-12-06 US US09/454,711 patent/US20030007963A1/en not_active Abandoned
- 1999-12-06 PT PT99965162T patent/PT1137762E/pt unknown
- 1999-12-06 JP JP2000586885A patent/JP4990434B2/ja not_active Expired - Lifetime
- 1999-12-06 EP EP99965162A patent/EP1137762B1/en not_active Revoked
- 1999-12-06 PT PT81648255T patent/PT2020438T/pt unknown
- 1999-12-06 WO PCT/US1999/029042 patent/WO2000034451A1/en not_active Application Discontinuation
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2003
- 2003-06-30 US US10/611,598 patent/US7351410B2/en not_active Expired - Lifetime
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2008
- 2008-01-30 US US12/012,003 patent/US7655226B2/en not_active Expired - Fee Related
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2009
- 2009-03-24 NL NL300382C patent/NL300382I2/nl unknown
- 2009-03-25 LU LU91544C patent/LU91544I2/fr unknown
- 2009-04-20 JP JP2009102515A patent/JP2009161562A/ja not_active Withdrawn
- 2009-12-09 US US12/633,932 patent/US20100092449A1/en not_active Abandoned
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2011
- 2011-12-21 US US13/333,052 patent/US20130039901A1/en not_active Abandoned
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2012
- 2012-08-17 JP JP2012181032A patent/JP2012224643A/ja not_active Withdrawn
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2013
- 2013-05-03 US US13/886,573 patent/US20140037611A1/en not_active Abandoned
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2015
- 2015-05-05 JP JP2015094511A patent/JP2015134836A/ja not_active Withdrawn
- 2015-09-23 US US14/863,333 patent/US20160243203A1/en not_active Abandoned
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2017
- 2017-01-04 JP JP2017000155A patent/JP2017066160A/ja not_active Withdrawn
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2018
- 2018-03-28 JP JP2018061663A patent/JP2018115200A/ja not_active Withdrawn
- 2018-07-10 CY CY20181100718T patent/CY1121113T1/el unknown
- 2018-07-25 CY CY2018020C patent/CY2018020I2/el unknown
- 2018-09-28 BE BE2018C038C patent/BE2018C038I2/fr unknown
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2019
- 2019-06-05 JP JP2019105319A patent/JP2019172685A/ja active Pending
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