CY1121113T1 - Θεραπεια της νοσου ρομρε - Google Patents

Θεραπεια της νοσου ρομρε

Info

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
Application number
CY20181100718T
Other languages
English (en)
Inventor
Johannes Gernardus Mathias Marie Van Bree
Edna Henriette Germaine Venneker
David P. Meeker
Original Assignee
Genzyme Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=22337648&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1121113(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1121113T1 publication Critical patent/CY1121113T1/el

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
    • 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
    • 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
    • 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
    • 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
    • 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)
    • 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
    • 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)
  • 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.
CY20181100718T 1998-12-07 2018-07-10 Θεραπεια της νοσου ρομρε CY1121113T1 (el)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
JP2009161562A (ja) 2009-07-23
JP2002531581A (ja) 2002-09-24
EP2020438A1 (en) 2009-02-04
NL300382I2 (en) 2009-12-01
US20140037611A1 (en) 2014-02-06
ATE410506T1 (de) 2008-10-15
DE122009000018I1 (de) 2009-09-17
KR20010101131A (ko) 2001-11-14
CA2353522A1 (en) 2000-06-15
PT2020438T (pt) 2018-07-13
PT1137762E (pt) 2009-01-14
US20040081645A1 (en) 2004-04-29
CY2018020I1 (el) 2019-07-10
JP2012224643A (ja) 2012-11-15
LU91544I2 (fr) 2009-05-25
US20030007963A1 (en) 2003-01-09
US20080206223A1 (en) 2008-08-28
US20160243203A1 (en) 2016-08-25
US7655226B2 (en) 2010-02-02
DK1137762T3 (da) 2009-02-02
EP1137762A1 (en) 2001-10-04
DK2020438T3 (en) 2018-07-23
DE69939705D1 (de) 2008-11-20
JP2015134836A (ja) 2015-07-27
ES2677343T3 (es) 2018-08-01
EP1137762A4 (en) 2005-01-12
US20130039901A1 (en) 2013-02-14
US20100092449A1 (en) 2010-04-15
ES2312222T3 (es) 2009-02-16
JP2018115200A (ja) 2018-07-26
JP2019172685A (ja) 2019-10-10
US7351410B2 (en) 2008-04-01
WO2000034451A1 (en) 2000-06-15
EP1137762B1 (en) 2008-10-08
EP2020438B1 (en) 2018-04-11
NL300382I1 (nl) 2009-06-02
CY2018020I2 (el) 2019-07-10
JP2017066160A (ja) 2017-04-06
BE2018C038I2 (el) 2019-03-05
AU3113700A (en) 2000-06-26
JP4990434B2 (ja) 2012-08-01

Similar Documents

Publication Publication Date Title
CY1121113T1 (el) Θεραπεια της νοσου ρομρε
CY1119369T1 (el) Ιατρικα παρασκευασματα για τη θεραπευτικη αγωγη της ανεπαρκειας της αλφα-γαλακτοσιδασης α
CY1118849T1 (el) Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
AP2000001785A0 (en) Medicaments
BR0309665A (pt) Tratamento de deficiência de alfa-galactosidase a
KR930702026A (ko) 자기항원의 경구 투여에 의한 자기면역 질병의 치료
WO2003096990A3 (en) Protein cages for the delivery of medical imaging and therapy
BR9913932A (pt) Uso de análogos de glp-1 no tratamento de derrame
GEP20094798B (en) (s,s)-reboxetine for treating chronic pain
EA200000747A1 (ru) СПОСОБ ВВЕДЕНИЯ AspB28-ИНСУЛИНА ЧЕЛОВЕКА
CY1105818T1 (el) Χρησεις του et743 στην θepαπευτικη αγωγη του καρκινου
EA200300544A1 (ru) Эффективные противоопухолевые лекарственные средства
DE69814394D1 (de) Verwendung von levobupivacain
NO994165L (no) Anvendelse av deskarboetoksyloratadin for fremstilling av et medikament for behandling av urininkontinens, reisesyke og svimmelhet
DK0668077T3 (da) Produkter, som indeholder G-CSF og TNF-bindende protein
MXPA00010254A (es) Tratamiento de disfuncion sexual en ciertos grupos de pacientes.
MD970254A (en) Use of melatonine for treatment of patients having a medicamental dependence
FI20020197A0 (fi) Yhdistelmä akuutin myokardiilisen infarktin hoitoon
ATE297732T1 (de) Kombinationstherapie zur behandlung von herzinsuffizienz
EA199901061A1 (ru) Новые соли врс-пептидов с органопротективной активностью, способ их получения и их использование в терапии
TR199901385T2 (xx) Piperidin t�revleri.
DE3374066D1 (en) N-acyl derivatives of peptides, their manufacture and use in combating diseases and means therefor
CY1108922T1 (el) Θεραπεια μελανωματος
DK1150981T3 (da) Terapeutisk necleosidforbindelse
GB9704046D0 (en) Arrestable therapeutic