NL300382I2 - Treatment of Pompe's disease - Google Patents

Treatment of Pompe's disease

Info

Publication number
NL300382I2
NL300382I2 NL300382C NL300382C NL300382I2 NL 300382 I2 NL300382 I2 NL 300382I2 NL 300382 C NL300382 C NL 300382C NL 300382 C NL300382 C NL 300382C NL 300382 I2 NL300382 I2 NL 300382I2
Authority
NL
Netherlands
Prior art keywords
disease
pompe
treatment
patient
body weight
Prior art date
Application number
NL300382C
Other languages
English (en)
Other versions
NL300382I1 (nl
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=NL300382(I2) "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 NL300382I1 publication Critical patent/NL300382I1/nl
Publication of NL300382I2 publication Critical patent/NL300382I2/nl

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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Husbandry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Obesity (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
NL300382C 1998-12-07 2009-03-24 Treatment of Pompe's disease NL300382I2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11129198P 1998-12-07 1998-12-07
PCT/US1999/029042 WO2000034451A1 (en) 1998-12-07 1999-12-06 Treatment of pompe's disease

Publications (2)

Publication Number Publication Date
NL300382I1 NL300382I1 (nl) 2009-06-02
NL300382I2 true NL300382I2 (en) 2009-12-01

Family

ID=22337648

Family Applications (1)

Application Number Title Priority Date Filing Date
NL300382C NL300382I2 (en) 1998-12-07 2009-03-24 Treatment of Pompe's disease

Country Status (16)

Country Link
US (7) US20030007963A1 (nl)
EP (2) EP2020438B1 (nl)
JP (7) JP4990434B2 (nl)
KR (1) KR20010101131A (nl)
AT (1) ATE410506T1 (nl)
AU (1) AU3113700A (nl)
BE (1) BE2018C038I2 (nl)
CA (1) CA2353522A1 (nl)
CY (2) CY1121113T1 (nl)
DE (2) DE122009000018I1 (nl)
DK (2) DK1137762T3 (nl)
ES (2) ES2677343T3 (nl)
LU (1) LU91544I2 (nl)
NL (1) NL300382I2 (nl)
PT (2) PT2020438T (nl)
WO (1) WO2000034451A1 (nl)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE410506T1 (de) * 1998-12-07 2008-10-15 Genzyme Corp Behandlung der pompeschen krankheit
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
EP1782825B1 (en) * 2000-07-18 2014-09-10 Duke University Treatment of glycogen storage diseases type II
DK3108895T3 (en) * 2000-07-18 2018-11-26 Univ Duke Treatment of glycogen storage disease 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
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1974752B1 (en) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) * 2001-04-30 2009-07-14 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
JP5433133B2 (ja) * 2003-01-22 2014-03-05 デューク・ユニヴァーシティ リソソームポリペプチドを発現するための改善された構築物
CA2814774C (en) 2003-01-31 2016-03-22 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
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
AU2007322123A1 (en) * 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
EP2279210B1 (en) 2008-05-07 2017-04-12 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
SI2318037T1 (sl) 2008-07-08 2015-07-31 Duke University Metoda za zdravljenje bolezni glikogenoze
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
SI2561069T1 (sl) 2010-04-23 2017-07-31 Alexion Pharmaceuticals, Inc. Encim za bolezen lizosomskega shranjevanja
MX2013000320A (es) 2010-06-25 2013-06-05 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa.
NZ702808A (en) 2010-06-25 2016-08-26 Shire Human Genetic Therapies Methods and compositions for cns delivery of iduronate-2-sulfatase
EP3964230A1 (en) 2010-06-25 2022-03-09 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a
PT3626257T (pt) 2010-06-25 2021-11-12 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
NZ702801A (en) 2010-06-25 2016-08-26 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
RU2626514C2 (ru) 2010-06-25 2017-07-28 Шир Хьюман Дженетик Терапис, Инк. Доставка к цнс лечебных агентов
KR20150038636A (ko) 2010-09-09 2015-04-08 시나게바 바이오파르마, 코포레이션 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
JP5992902B2 (ja) * 2011-03-16 2016-09-14 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
US20140186326A1 (en) 2011-04-22 2014-07-03 William Canfield 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
AU2014218854B2 (en) 2013-02-20 2019-01-24 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
PT3201320T (pt) 2014-09-30 2024-01-12 Amicus Therapeutics Inc Alfa-glucosidase ácida altamente potente com hidratos de carbono intensificados
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
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
KR102444612B1 (ko) 2016-03-30 2022-09-21 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
KR20240001721A (ko) 2016-09-12 2024-01-03 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
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
JP2022540632A (ja) 2019-07-09 2022-09-16 ジェネトン 糖原病（ｇｓｄ）の処置

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
EP0502976B1 (en) 1989-12-01 1996-07-03 Pharming B.V. Production of recombinant polypeptides by bovine species and transgenic methods
ATE352612T1 (de) 1990-08-29 2007-02-15 Pharming Intellectual Pty Bv Homologe rekombination in säugetier-zellen
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
AU4537393A (en) * 1992-06-15 1994-01-04 Gene Pharming Europe Bv Production of recombinant polypeptides by bovine species and transgenic methods
EP0625530A1 (en) * 1993-05-17 1994-11-23 Nippon Paint Co., Ltd. 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
ATE410506T1 (de) * 1998-12-07 2008-10-15 Genzyme Corp Behandlung der pompeschen krankheit

Also Published As

Publication number Publication date
CY2018020I1 (el) 2019-07-10
EP1137762A1 (en) 2001-10-04
JP2002531581A (ja) 2002-09-24
ES2677343T3 (es) 2018-08-01
CY2018020I2 (el) 2019-07-10
EP1137762B1 (en) 2008-10-08
ES2312222T3 (es) 2009-02-16
US20100092449A1 (en) 2010-04-15
EP2020438A1 (en) 2009-02-04
KR20010101131A (ko) 2001-11-14
JP2012224643A (ja) 2012-11-15
JP2015134836A (ja) 2015-07-27
US7655226B2 (en) 2010-02-02
AU3113700A (en) 2000-06-26
DK1137762T3 (da) 2009-02-02
CA2353522A1 (en) 2000-06-15
NL300382I1 (nl) 2009-06-02
DK2020438T3 (en) 2018-07-23
JP2017066160A (ja) 2017-04-06
US20040081645A1 (en) 2004-04-29
PT2020438T (pt) 2018-07-13
WO2000034451A1 (en) 2000-06-15
EP1137762A4 (en) 2005-01-12
JP2019172685A (ja) 2019-10-10
DE122009000018I1 (de) 2009-09-17
ATE410506T1 (de) 2008-10-15
US20140037611A1 (en) 2014-02-06
JP2018115200A (ja) 2018-07-26
CY1121113T1 (el) 2019-07-10
DE69939705D1 (de) 2008-11-20
JP4990434B2 (ja) 2012-08-01
US20080206223A1 (en) 2008-08-28
LU91544I2 (fr) 2009-05-25
US7351410B2 (en) 2008-04-01
PT1137762E (pt) 2009-01-14
BE2018C038I2 (nl) 2019-03-05
EP2020438B1 (en) 2018-04-11
US20030007963A1 (en) 2003-01-09
US20130039901A1 (en) 2013-02-14
JP2009161562A (ja) 2009-07-23
US20160243203A1 (en) 2016-08-25

Similar Documents

Publication Publication Date Title
BE2018C038I2 (nl)
ZA883602B (en) Medicament
NZ514077A (en) Treatment of Α-galactosidase A deficiency
GR3030867T3 (en) Guanidine derivatives useful in therapy
NZ504026A (en) Methods for treating hypercoagulable states or acquired protein C deficiency using human-activated protein C
PL309594A1 (en) Apllication of rilusole in treating parkinson disease and parkinsonian syndromes
ZA95679B (en) Products containing G-CSF and TNF binding protein
BG104797A (en) Use of dexmedetomi dine for sedative effect in patients in an intensive care unit
EP1006793A4 (en) COMPOSITIONS FOR TREATING ARRHYTHMIA AND METHODS OF TREATMENT
EP0437929A3 (en) Use of diadenosine-5',5'''-p1,p4-tetraphosphate for the manufacture of a medicament for the treatment of heart disease
UA41329C2 (uk) Фармацевтична композиція для лікування порушень серцевого ритму у великих ссавців та людей, похідні 3-фенілсульфоніл-3,7-діазабіцикло[3,3,1]нонану та лікарський засіб для лікування аритмії
GR3032224T3 (en) Therapeutic agent for threatened abortion.
IL118833A (en) Sertraline for use as a medicament in the treatment of post-myocardial infarction patients
EP0642338A1 (en) USE OF (E) - 2 - (P -FLUOROPHENETHYL) - 3 - FLUOROALLYLAMINE FOR TREATING ALZHEIMER'S DISEASE.
NZ268039A (en) Use of efaroxan in treating parkinson's disease
ZA941816B (en) Benzimidazole derivatives their preparation and use
EP0353910A3 (en) Human interferon-gamma, process for preparing said human interferon-gamma, and its use
AU616657B2 (en) Butyl hydroxyanisoles for the treatment of retroviral diseases
UA10301A (uk) Спосіб лікуваhhя раку шлуhку
MD1469G2 (ro) Metodă de tratament al şocului combustional
UA22223A (uk) Спосіб лікуваhhя захворюваhь оргаhа зору
UA36521A (uk) Спосіб здійснення реабілітаційно-оздоровчих технологій, що передбачають корегування функціонального стану організму пацієнта проведенням процедур біоадаптивного керування з використанням біологічного зворотного зв'язку,та апаратний комплекс для його здій