PT1137762E - Tratamento da doença de pompe - Google Patents

Tratamento da doença de pompe Download PDF

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Publication number
PT1137762E
PT1137762E PT99965162T PT99965162T PT1137762E PT 1137762 E PT1137762 E PT 1137762E PT 99965162 T PT99965162 T PT 99965162T PT 99965162 T PT99965162 T PT 99965162T PT 1137762 E PT1137762 E PT 1137762E
Authority
PT
Portugal
Prior art keywords
disease
pompe
treatment
patient
body weight
Prior art date
Application number
PT99965162T
Other languages
English (en)
Inventor
Johannes Brenardus Mathias Marie Van Bree
Edna Henriette Germaine Venneker
David P Meeker
Original Assignee
Genzyme Corp
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=PT1137762(E) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of PT1137762E publication Critical patent/PT1137762E/pt

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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

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  • 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)
PT99965162T 1998-12-07 1999-12-06 Tratamento da doença de pompe PT1137762E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US11129198P 1998-12-07 1998-12-07

Publications (1)

Publication Number Publication Date
PT1137762E true PT1137762E (pt) 2009-01-14

Family

ID=22337648

Family Applications (2)

Application Number Title Priority Date Filing Date
PT81648255T PT2020438T (pt) 1998-12-07 1999-12-06 Tratamento da doença de pompe
PT99965162T PT1137762E (pt) 1998-12-07 1999-12-06 Tratamento da doença de pompe

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT81648255T PT2020438T (pt) 1998-12-07 1999-12-06 Tratamento da doença de pompe

Country Status (16)

Country Link
US (7) US20030007963A1 (pt)
EP (2) EP2020438B1 (pt)
JP (7) JP4990434B2 (pt)
KR (1) KR20010101131A (pt)
AT (1) ATE410506T1 (pt)
AU (1) AU3113700A (pt)
BE (1) BE2018C038I2 (pt)
CA (1) CA2353522A1 (pt)
CY (2) CY1121113T1 (pt)
DE (2) DE122009000018I1 (pt)
DK (2) DK1137762T3 (pt)
ES (2) ES2312222T3 (pt)
LU (1) LU91544I2 (pt)
NL (1) NL300382I2 (pt)
PT (2) PT2020438T (pt)
WO (1) WO2000034451A1 (pt)

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DK1137762T3 (da) * 1998-12-07 2009-02-02 Genzyme Corp Behandling af Pompes sygdom
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AU2016256663A1 (en) * 2000-07-18 2016-11-24 Duke University Treatment of clycogen storage disease type II
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US20050244400A1 (en) * 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
KR100697270B1 (ko) * 2004-12-10 2007-03-21 삼성전자주식회사 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법
KR20080025373A (ko) 2005-05-17 2008-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 1-데옥시노지리마이신 및 유도체를 이용한 폼페병의 치료방법
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
WO2008063511A2 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
EP2279210B1 (en) 2008-05-07 2017-04-12 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
EP2318037B1 (en) 2008-07-08 2015-01-28 Duke University Method of treating glycogen storage disease
EP2475376B1 (en) * 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
US8536148B2 (en) 2009-09-04 2013-09-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Disabling autophagy as a treatment for lysosomal storage diseases
PT3205351T (pt) 2010-04-23 2023-06-28 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
KR102537084B1 (ko) 2010-06-25 2023-05-26 샤이어 휴먼 지네틱 테라피즈 인크. 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
KR20130043166A (ko) 2010-06-25 2013-04-29 샤이어 휴먼 지네틱 테라피즈 인크. 산필리포 증후군 b형의 치료
ES2895655T3 (es) 2010-06-25 2022-02-22 Shire Human Genetic Therapies Métodos y composiciones para la administración al SNC de iduronato-2-sulfatasa
CN103260637B (zh) 2010-06-25 2016-04-06 夏尔人类遗传性治疗公司 乙酰肝素n-硫酸酯酶cns递送的方法和组合物
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
EP2593131B1 (en) 2010-06-25 2019-08-21 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
CN105457018A (zh) 2010-09-09 2016-04-06 辛那杰瓦生物制药股份有限公司 使用溶酶体酸性脂肪酶来治疗患者的溶酶体酸性脂肪酶缺乏
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
EP2687597B1 (en) * 2011-03-16 2018-12-26 Amano Enzyme Inc. Modified alpha-glucosidase and applications of same
CN103797115A (zh) * 2011-04-22 2014-05-14 建新公司 具有快速加工性能的经过修饰的酸性α葡糖苷酶
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
CA2901978A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
MA40460A (fr) 2014-08-11 2016-02-18 Shire Human Genetic Therapies Ciblage lysosomial et utilisation correspondante
AU2015325028B2 (en) 2014-09-30 2022-02-24 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
EP3344337A4 (en) * 2015-08-31 2019-03-06 Duke University METHOD AND COMPOSITIONS FOR TREATING CYTOPLASMATIC GLYCOGEN MEMORY DISEASES
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EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
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JP2022540632A (ja) 2019-07-09 2022-09-16 ジェネトン 糖原病（ｇｓｄ）の処置

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Also Published As

Publication number Publication date
CY1121113T1 (el) 2019-07-10
JP2015134836A (ja) 2015-07-27
ES2312222T3 (es) 2009-02-16
US20160243203A1 (en) 2016-08-25
EP1137762A1 (en) 2001-10-04
US20140037611A1 (en) 2014-02-06
US7655226B2 (en) 2010-02-02
LU91544I2 (fr) 2009-05-25
US20030007963A1 (en) 2003-01-09
BE2018C038I2 (pt) 2019-03-05
JP2002531581A (ja) 2002-09-24
AU3113700A (en) 2000-06-26
EP1137762B1 (en) 2008-10-08
JP2012224643A (ja) 2012-11-15
US7351410B2 (en) 2008-04-01
ES2677343T3 (es) 2018-08-01
JP4990434B2 (ja) 2012-08-01
PT2020438T (pt) 2018-07-13
US20100092449A1 (en) 2010-04-15
US20130039901A1 (en) 2013-02-14
CA2353522A1 (en) 2000-06-15
CY2018020I2 (el) 2019-07-10
DE122009000018I1 (de) 2009-09-17
EP2020438B1 (en) 2018-04-11
DK1137762T3 (da) 2009-02-02
JP2017066160A (ja) 2017-04-06
CY2018020I1 (el) 2019-07-10
DK2020438T3 (en) 2018-07-23
JP2009161562A (ja) 2009-07-23
NL300382I2 (en) 2009-12-01
WO2000034451A1 (en) 2000-06-15
US20040081645A1 (en) 2004-04-29
EP2020438A1 (en) 2009-02-04
KR20010101131A (ko) 2001-11-14
JP2018115200A (ja) 2018-07-26
EP1137762A4 (en) 2005-01-12
JP2019172685A (ja) 2019-10-10
US20080206223A1 (en) 2008-08-28
DE69939705D1 (de) 2008-11-20
ATE410506T1 (de) 2008-10-15
NL300382I1 (nl) 2009-06-02

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