JP2010509344A - ポンペ病を治療するための方法 - Google Patents

ポンペ病を治療するための方法 Download PDF

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Publication number
JP2010509344A
JP2010509344A JP2009536345A JP2009536345A JP2010509344A JP 2010509344 A JP2010509344 A JP 2010509344A JP 2009536345 A JP2009536345 A JP 2009536345A JP 2009536345 A JP2009536345 A JP 2009536345A JP 2010509344 A JP2010509344 A JP 2010509344A
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JP
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gaa
human
fusion protein
targeting domain
mannose
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JP2009536345A
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Japanese (ja)
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JP2010509344A5 (enExample
Inventor
ジョナサン レボウィッツ,
ジョン マーガ,
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ザイストール セラピューティクス, インコーポレイテッド
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Publication of JP2010509344A publication Critical patent/JP2010509344A/ja
Publication of JP2010509344A5 publication Critical patent/JP2010509344A5/ja
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2009536345A 2006-11-13 2007-11-13 ポンペ病を治療するための方法 Pending JP2010509344A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US85851406P 2006-11-13 2006-11-13
US87925507P 2007-01-05 2007-01-05
US90018707P 2007-02-07 2007-02-07
PCT/US2007/023881 WO2008063511A2 (en) 2006-11-13 2007-11-13 Methods for treating pompe disease

Publications (2)

Publication Number Publication Date
JP2010509344A true JP2010509344A (ja) 2010-03-25
JP2010509344A5 JP2010509344A5 (enExample) 2011-06-23

Family

ID=39333078

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009536345A Pending JP2010509344A (ja) 2006-11-13 2007-11-13 ポンペ病を治療するための方法

Country Status (7)

Country Link
US (2) US20090117091A1 (enExample)
EP (1) EP2099523A2 (enExample)
JP (1) JP2010509344A (enExample)
AU (1) AU2007322123A1 (enExample)
CA (1) CA2669347A1 (enExample)
IL (1) IL198692A0 (enExample)
WO (1) WO2008063511A2 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014513952A (ja) * 2011-04-22 2014-06-19 ジェンザイム・コーポレーション 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 Jcrファーマ株式会社 マンノース-6-リン酸の数を減少させた糖蛋白質

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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
WO2012061597A1 (en) * 2010-11-03 2012-05-10 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer, National Institutes Of Health Alpha-glucosidase binders and methods of their use
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
DK3201320T5 (da) 2014-09-30 2024-10-14 Amicus Therapeutics Inc Højpotent sur alfa-glucosidase med forbedrede carbohydrater
MX394968B (es) 2015-12-30 2025-03-24 Amicus Therapeutics Inc Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
KR102343162B1 (ko) 2016-03-30 2021-12-23 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
BR112020021962A2 (pt) 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
MX2020012350A (es) 2018-05-17 2021-01-29 Regeneron Pharma Anticuerpos anti-cd63, conjugados y usos de estos.
EP3863663A2 (en) 2018-10-10 2021-08-18 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
BR112021021720A2 (pt) * 2019-04-30 2021-12-28 Univ Pennsylvania Composições úteis para tratamento de doença de pompe
EP3871687A1 (en) 2020-02-27 2021-09-01 eleva GmbH Enzyme replacement therapy for treating pompe disease
WO2021231863A1 (en) * 2020-05-14 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
WO2025137213A1 (en) 2023-12-20 2025-06-26 Denali Therapeutics Inc. Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014513952A (ja) * 2011-04-22 2014-06-19 ジェンザイム・コーポレーション 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 Jcrファーマ株式会社 マンノース-6-リン酸の数を減少させた糖蛋白質

Also Published As

Publication number Publication date
WO2008063511A3 (en) 2009-04-09
WO2008063511A2 (en) 2008-05-29
EP2099523A2 (en) 2009-09-16
AU2007322123A1 (en) 2008-05-29
US20120093794A1 (en) 2012-04-19
CA2669347A1 (en) 2008-05-29
US20090117091A1 (en) 2009-05-07
IL198692A0 (en) 2011-07-31

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