BE2018C038I2 - - Google Patents
Info
- Publication number
- BE2018C038I2 BE2018C038I2 BE2018C038C BE2018C038C BE2018C038I2 BE 2018C038 I2 BE2018C038 I2 BE 2018C038I2 BE 2018C038 C BE2018C038 C BE 2018C038C BE 2018C038 C BE2018C038 C BE 2018C038C BE 2018C038 I2 BE2018C038 I2 BE 2018C038I2
- Authority
- BE
- Belgium
- Prior art keywords
- disease
- patient
- body weight
- provides methods
- comprises administering
- Prior art date
Links
- 206010053185 Glycogen storage disease type II Diseases 0.000 abstract 1
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 abstract 1
- 230000037396 body weight Effects 0.000 abstract 1
- 201000004502 glycogen storage disease II Diseases 0.000 abstract 1
- 102000045921 human GAA Human genes 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- 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
-
- 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)
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biodiversity & Conservation Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Animal Husbandry (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Cardiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Obesity (AREA)
- Dermatology (AREA)
- Heart & Thoracic Surgery (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US11129198P | 1998-12-07 | 1998-12-07 | |
EP99965162A EP1137762B1 (fr) | 1998-12-07 | 1999-12-06 | Traitement de la maladie de pompe |
Publications (1)
Publication Number | Publication Date |
---|---|
BE2018C038I2 true BE2018C038I2 (fr) | 2019-03-05 |
Family
ID=22337648
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BE2018C038C BE2018C038I2 (fr) | 1998-12-07 | 2018-09-28 |
Country Status (16)
Country | Link |
---|---|
US (7) | US20030007963A1 (fr) |
EP (2) | EP1137762B1 (fr) |
JP (7) | JP4990434B2 (fr) |
KR (1) | KR20010101131A (fr) |
AT (1) | ATE410506T1 (fr) |
AU (1) | AU3113700A (fr) |
BE (1) | BE2018C038I2 (fr) |
CA (1) | CA2353522A1 (fr) |
CY (2) | CY1121113T1 (fr) |
DE (2) | DE122009000018I1 (fr) |
DK (2) | DK1137762T3 (fr) |
ES (2) | ES2677343T3 (fr) |
LU (1) | LU91544I2 (fr) |
NL (1) | NL300382I2 (fr) |
PT (2) | PT1137762E (fr) |
WO (1) | WO2000034451A1 (fr) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2000034451A1 (fr) * | 1998-12-07 | 2000-06-15 | Pharming Intellectual Property B.V. | Traitement de la maladie de pompe |
BR0108077A (pt) * | 2000-02-04 | 2002-10-22 | Children S Hospital Res Founda | Processo para reduzir placas ateroscleróticas em um mamìfero, método para tratamento da aterosclerose em um mamìfero, composição, métodos para prover proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo ou mistura deles, para células de um mamìfero tendo deficiência na proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo , para prover lipase de ácido lisossomal biologicamente ativa para células de um mamìfero tendo deficiência de lipase de ácido lisossomal biologicamente ativa, para prover lipase de ácido lisossoal biologicamente ativa para células de um mamìfero com aterosclerose, para tratamento da doença de wolman em um mamìfero, para tratamento de doença de armazenagem de colesteril éster em um mamìfero, e, para tratamento da aterosclerose em um mamìfero |
AU2016256663A1 (en) * | 2000-07-18 | 2016-11-24 | Duke University | Treatment of clycogen storage disease type II |
DK3108895T3 (en) | 2000-07-18 | 2018-11-26 | Univ Duke | Treatment of glycogen storage disease type II |
EP1782825B1 (fr) * | 2000-07-18 | 2014-09-10 | Duke University | Traitement d'une glycogénose de 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 |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
ATE384736T1 (de) * | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
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 |
WO2004064750A2 (fr) | 2003-01-22 | 2004-08-05 | Duke University | Constructions ameliorees permettant d'exprimer des polypeptides lysosomiaux |
CA2814774C (fr) * | 2003-01-31 | 2016-03-22 | Mount Sinai School Of Medicine Of New York University | Therapie combinee destinee au traitement de troubles associes a une carence proteique |
JP4914224B2 (ja) * | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | 酸性αグルコシダーゼおよびそのフラグメント |
KR100697270B1 (ko) * | 2004-12-10 | 2007-03-21 | 삼성전자주식회사 | 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법 |
EP3782655A1 (fr) | 2005-05-17 | 2021-02-24 | Amicus Therapeutics, Inc. | Procédé pour le traitement de la maladie de pompe au moyen de 1-désoxynojirimycine et de ses dérivés |
WO2007030375A2 (fr) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Therapie a base de lipase acide lysosomiale destinee au traitement de la steatose hepatique non alcoolique et de maladies associees |
WO2008063511A2 (fr) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Méthodes de traitement de la maladie de pompe |
AU2009244148B2 (en) | 2008-05-07 | 2014-10-09 | Biomarin Pharmaceutical Inc. | Lysosomal targeting peptides and uses thereof |
PT2318037E (pt) | 2008-07-08 | 2015-05-20 | Univ Duke | Método de tratamento da doença de armazenamento de glicogénio |
EP3679942A1 (fr) | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulations pour des enzymes lysosomales |
WO2011028941A2 (fr) | 2009-09-04 | 2011-03-10 | The United States Of America, As Represented By The Secretary Department Of Health & Human Services | Inhibition de l'autophagie à titre de traitement pour les maladies de surcharge lysosomales |
EP4241854A3 (fr) | 2010-04-23 | 2023-11-08 | Alexion Pharmaceuticals, Inc. | Enzyme des maladies lysosomales |
AU2011270668B8 (en) | 2010-06-25 | 2017-02-09 | Takeda Pharmaceutical Company Limited | CNS delivery of therapeutic agents |
PT2593131T (pt) | 2010-06-25 | 2019-10-30 | Shire Human Genetic Therapies | Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase |
CA2805413A1 (fr) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Procedes et compositions pour une administration au snc d'heparane n-sulfatase |
DK2585104T3 (da) | 2010-06-25 | 2019-10-14 | Shire Human Genetic Therapies | Fremgangsmåder og sammensætninger tilcns-levering af arylsulfatase a |
AU2011270672B2 (en) | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
KR102151889B1 (ko) | 2010-06-25 | 2020-09-04 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
PL2977057T3 (pl) | 2010-09-09 | 2020-06-01 | Alexion Pharmaceuticals, Inc. | Izolowana rekombinowana ludzka lizosomalna N-glikozylowana kwaśna lipaza |
KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
WO2012124520A1 (fr) * | 2011-03-16 | 2012-09-20 | 天野エンザイム株式会社 | Alpha-glucosidase modifiée et ses applications |
PE20140617A1 (es) | 2011-04-22 | 2014-05-28 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado |
EP2793922B1 (fr) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Formulations stables pour l'administration au système nerveux central d'arylsulfatase a |
KR20230066482A (ko) | 2012-03-07 | 2023-05-15 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
WO2014130723A1 (fr) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Méthodes et compositions pour le traitement de la maladie de pompe |
US10603364B2 (en) | 2014-08-11 | 2020-03-31 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
CN107075468B (zh) | 2014-09-30 | 2021-12-24 | 阿米库斯治疗学公司 | 具有增强的碳水化合物的高强度酸性α-葡糖苷酶 |
MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
WO2017040647A1 (fr) * | 2015-08-31 | 2017-03-09 | Duke University | Méthodes et compositions pour le traitement de troubles associés à une accumulation du glycogène cytoplasmique |
US10940125B2 (en) | 2015-09-18 | 2021-03-09 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
WO2017117407A1 (fr) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Alpha-glucosidase acide améliorée pour le traitement de la maladie de pompe |
CA3019128A1 (fr) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprenant une alpha-glucosidase acide recombinante |
IL295551A (en) | 2016-03-30 | 2022-10-01 | Amicus Therapeutics Inc | A method for selecting recombinant proteins with a high amount of mannose-6-phosphate |
WO2018046774A1 (fr) | 2016-09-12 | 2018-03-15 | Genethon | Variants de l'acide alpha-glucosidase et leurs utilisations |
EP3293260A1 (fr) | 2016-09-12 | 2018-03-14 | Genethon | Variants d'alpha-glucosidase acide et leurs utilisations |
EP3293203A1 (fr) | 2016-09-12 | 2018-03-14 | Genethon | Variants d'alpha-glucosidase acide et leurs utilisations |
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 (fr) * | 1987-02-17 | 1998-04-01 | Pharming B.V. | Séquences d'ADN pour diriger des protéines vers les glandes mammaires, afin d'être secrétées efficacement |
CA2351200A1 (fr) | 1989-12-01 | 1991-06-13 | Gene Pharming Europe Bv | Production de polypeptides recombinant par especes bovines et procedes transgeniques |
CA2090473A1 (fr) | 1990-08-29 | 1992-03-01 | Robert M. Kay | Recombinaison homologue dans des cellules mammaliennes |
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 |
EP0652889A4 (fr) * | 1992-06-15 | 1997-05-07 | Gene Pharming Europ Bv | Production de polypeptides recombines par des especes bovines et des procedes transgeniques. |
TW319781B (fr) * | 1993-05-17 | 1997-11-11 | Nippon Painting Co Ltd | |
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 |
WO2000034451A1 (fr) * | 1998-12-07 | 2000-06-15 | Pharming Intellectual Property B.V. | Traitement de la maladie de pompe |
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1999
- 1999-12-06 WO PCT/US1999/029042 patent/WO2000034451A1/fr not_active Application Discontinuation
- 1999-12-06 DK DK99965162T patent/DK1137762T3/da active
- 1999-12-06 EP EP99965162A patent/EP1137762B1/fr not_active Revoked
- 1999-12-06 ES ES08164825.5T patent/ES2677343T3/es not_active Expired - Lifetime
- 1999-12-06 JP JP2000586885A patent/JP4990434B2/ja not_active Expired - Lifetime
- 1999-12-06 AU AU31137/00A patent/AU3113700A/en not_active Abandoned
- 1999-12-06 DE DE122009000018C patent/DE122009000018I1/de active Pending
- 1999-12-06 ES ES99965162T patent/ES2312222T3/es not_active Expired - Lifetime
- 1999-12-06 DE DE69939705T patent/DE69939705D1/de not_active Expired - Lifetime
- 1999-12-06 DK DK08164825.5T patent/DK2020438T3/en active
- 1999-12-06 EP EP08164825.5A patent/EP2020438B1/fr not_active Revoked
- 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 CA CA002353522A patent/CA2353522A1/fr not_active Abandoned
- 1999-12-06 PT PT81648255T patent/PT2020438T/pt unknown
- 1999-12-06 AT AT99965162T patent/ATE410506T1/de active
- 1999-12-06 KR KR1020017007040A patent/KR20010101131A/ko 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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