JP2002531581A5 - - Google Patents

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Publication number
JP2002531581A5
JP2002531581A5 JP2000586885A JP2000586885A JP2002531581A5 JP 2002531581 A5 JP2002531581 A5 JP 2002531581A5 JP 2000586885 A JP2000586885 A JP 2000586885A JP 2000586885 A JP2000586885 A JP 2000586885A JP 2002531581 A5 JP2002531581 A5 JP 2002531581A5
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JP
Japan
Prior art keywords
pharmaceutical composition
dosage
composition according
glucosidase
patient
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2000586885A
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English (en)
Japanese (ja)
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JP2002531581A (ja
JP4990434B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US1999/029042 external-priority patent/WO2000034451A1/en
Publication of JP2002531581A publication Critical patent/JP2002531581A/ja
Publication of JP2002531581A5 publication Critical patent/JP2002531581A5/ja
Application granted granted Critical
Publication of JP4990434B2 publication Critical patent/JP4990434B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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JP2000586885A 1998-12-07 1999-12-06 ポンペ病の処置 Expired - Lifetime JP4990434B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11129198P 1998-12-07 1998-12-07
US60/111,291 1998-12-07
PCT/US1999/029042 WO2000034451A1 (en) 1998-12-07 1999-12-06 Treatment of pompe's disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2009102515A Division JP2009161562A (ja) 1998-12-07 2009-04-20 ポンペ病の処置

Publications (3)

Publication Number Publication Date
JP2002531581A JP2002531581A (ja) 2002-09-24
JP2002531581A5 true JP2002531581A5 (https=) 2006-02-02
JP4990434B2 JP4990434B2 (ja) 2012-08-01

Family

ID=22337648

Family Applications (7)

Application Number Title Priority Date Filing Date
JP2000586885A Expired - Lifetime JP4990434B2 (ja) 1998-12-07 1999-12-06 ポンペ病の処置
JP2009102515A Withdrawn JP2009161562A (ja) 1998-12-07 2009-04-20 ポンペ病の処置
JP2012181032A Withdrawn JP2012224643A (ja) 1998-12-07 2012-08-17 ポンペ病の処置
JP2015094511A Withdrawn JP2015134836A (ja) 1998-12-07 2015-05-05 ポンペ病の処置
JP2017000155A Withdrawn JP2017066160A (ja) 1998-12-07 2017-01-04 ポンペ病の処置
JP2018061663A Withdrawn JP2018115200A (ja) 1998-12-07 2018-03-28 ポンペ病の処置
JP2019105319A Pending JP2019172685A (ja) 1998-12-07 2019-06-05 ポンペ病の処置

Family Applications After (6)

Application Number Title Priority Date Filing Date
JP2009102515A Withdrawn JP2009161562A (ja) 1998-12-07 2009-04-20 ポンペ病の処置
JP2012181032A Withdrawn JP2012224643A (ja) 1998-12-07 2012-08-17 ポンペ病の処置
JP2015094511A Withdrawn JP2015134836A (ja) 1998-12-07 2015-05-05 ポンペ病の処置
JP2017000155A Withdrawn JP2017066160A (ja) 1998-12-07 2017-01-04 ポンペ病の処置
JP2018061663A Withdrawn JP2018115200A (ja) 1998-12-07 2018-03-28 ポンペ病の処置
JP2019105319A Pending JP2019172685A (ja) 1998-12-07 2019-06-05 ポンペ病の処置

Country Status (16)

Country Link
US (7) US20030007963A1 (https=)
EP (2) EP1137762B1 (https=)
JP (7) JP4990434B2 (https=)
KR (1) KR20010101131A (https=)
AT (1) ATE410506T1 (https=)
AU (1) AU3113700A (https=)
BE (1) BE2018C038I2 (https=)
CA (1) CA2353522A1 (https=)
CY (2) CY1121113T1 (https=)
DE (2) DE69939705D1 (https=)
DK (2) DK1137762T3 (https=)
ES (2) ES2312222T3 (https=)
LU (1) LU91544I2 (https=)
NL (1) NL300382I2 (https=)
PT (2) PT1137762E (https=)
WO (1) WO2000034451A1 (https=)

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DK1137762T3 (da) * 1998-12-07 2009-02-02 Genzyme Corp Behandling af Pompes sygdom
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
AU7194101A (en) * 2000-07-18 2002-01-30 Univ Duke 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
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
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
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, 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
AU2003224880A1 (en) 2002-04-05 2003-10-27 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
PT2441467E (pt) * 2003-01-31 2015-10-12 Sinai School Medicine Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica
CN1922313B (zh) * 2004-02-10 2011-10-26 生物马林医药公司 酸性α-糖苷酶及其片段
KR100697270B1 (ko) * 2004-12-10 2007-03-21 삼성전자주식회사 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법
EP2932982B1 (en) 2005-05-17 2018-10-03 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
US20090117091A1 (en) * 2006-11-13 2009-05-07 Lebowitz Jonathan Methods for treating pompe disease
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
ES2535461T3 (es) * 2008-07-08 2015-05-11 Duke University Método de tratamiento de la enfermedad por almacenamiento de glucógeno
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
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
PT2561069T (pt) 2010-04-23 2017-05-22 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
CN103179980B (zh) 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
CA2805449A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
CA2810999C (en) 2010-09-09 2023-10-03 Anthony Quinn Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
DK2687597T3 (en) * 2011-03-16 2019-02-25 Amano Enzyme Inc MODIFIED ALPHA-GLUCOSIDASE AND USES OF SAME
MX2013012345A (es) 2011-04-22 2015-05-07 Genzyme Corp Alfa glucosidasa acida modificada con procesamiento acelerado.
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
EP2981551B1 (en) 2013-02-20 2020-06-03 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
WO2016054231A1 (en) 2014-09-30 2016-04-07 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
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
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
US10227577B2 (en) * 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
MX2019002842A (es) 2016-09-12 2019-08-29 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas.
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
CA3155432A1 (en) * 2019-12-17 2021-06-24 Navigo Proteins Gmbh Binding proteins for the enzyme acid alpha glucosidase (gaa) and uses thereof

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EP0279582A3 (en) 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
CA2075206C (en) 1989-12-01 2006-05-23 Herbert L. Heyneker 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
EP0652889A4 (en) * 1992-06-15 1997-05-07 Gene Pharming Europ Bv PRODUCTION OF RECOMBINANT POLYPEPTIDES BY BOVINE SPECIES AND TRANSGENIC PROCESSES.
US5446077A (en) * 1993-05-17 1995-08-29 Nippon Paint Company, 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
DK1137762T3 (da) * 1998-12-07 2009-02-02 Genzyme Corp Behandling af Pompes sygdom

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