CR20170107A - Alfa-glucosidasa ácida muy potente con hidratos de carbono potenciados - Google Patents

Alfa-glucosidasa ácida muy potente con hidratos de carbono potenciados

Info

Publication number
CR20170107A
CR20170107A CR20170107A CR20170107A CR20170107A CR 20170107 A CR20170107 A CR 20170107A CR 20170107 A CR20170107 A CR 20170107A CR 20170107 A CR20170107 A CR 20170107A CR 20170107 A CR20170107 A CR 20170107A
Authority
CR
Costa Rica
Prior art keywords
glucosidase
potentiated
carbon hydrates
rhgaa
powerful acid
Prior art date
Application number
CR20170107A
Other languages
English (en)
Inventor
Hung Do
Russell Gotschall
Original Assignee
Amicus Therapeutics Inc
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
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CR20170107A publication Critical patent/CR20170107A/es

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    • 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)
    • 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)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • 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
    • 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)
    • 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
    • C12N2510/00—Genetically modified cells
    • C12N2510/02—Cells for production

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Emergency Medicine (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Composición de alfa glucosidasa humana recombinante (rhGAA) derivada de células CHO que contiene una composición de glicano más optimizada que consiste en una cantidad de rhGAA que contiene N-glicanos que llevan manosa-6-fosfato (M6P) o bis-M6P mayor que las rhGAAs convencionales, junto con una baja cantidad de glicanos de alto contenido en manosa no fosforilados y una baja cantidad de galactosa terminal en oligosacáridos complejos.
CR20170107A 2014-09-30 2015-09-30 Alfa-glucosidasa ácida muy potente con hidratos de carbono potenciados CR20170107A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462057842P 2014-09-30 2014-09-30
US201462057847P 2014-09-30 2014-09-30
US201562112463P 2015-02-05 2015-02-05
US201562135345P 2015-03-19 2015-03-19
PCT/US2015/053252 WO2016054231A1 (en) 2014-09-30 2015-09-30 Highly potent acid alpha-glucosidase with enhanced carbohydrates

Publications (1)

Publication Number Publication Date
CR20170107A true CR20170107A (es) 2017-09-01

Family

ID=55631441

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20170107A CR20170107A (es) 2014-09-30 2015-09-30 Alfa-glucosidasa ácida muy potente con hidratos de carbono potenciados

Country Status (41)

Country Link
US (6) US10208299B2 (es)
EP (3) EP4273241B1 (es)
JP (4) JP6851964B2 (es)
KR (4) KR102741048B1 (es)
CN (2) CN114540327B (es)
AU (3) AU2015325028B2 (es)
BR (1) BR112017005810A2 (es)
CA (2) CA3228032A1 (es)
CL (1) CL2017000722A1 (es)
CO (1) CO2017002776A2 (es)
CR (1) CR20170107A (es)
CY (2) CY1126544T1 (es)
DK (2) DK3201320T5 (es)
DO (1) DOP2017000079A (es)
EA (1) EA038986B1 (es)
EC (1) ECSP17019103A (es)
ES (2) ES3017134T3 (es)
FI (4) FI3201320T3 (es)
FR (2) FR24C1012I1 (es)
HR (2) HRP20250058T1 (es)
HU (4) HUE070443T2 (es)
IL (4) IL307587A (es)
LT (4) LT4273241T (es)
LU (1) LUC00336I2 (es)
MX (2) MX388241B (es)
MY (1) MY186336A (es)
NI (1) NI201700039A (es)
NO (1) NO2025023I1 (es)
PE (1) PE20170906A1 (es)
PH (1) PH12017500455A1 (es)
PL (2) PL3201320T3 (es)
PT (2) PT3201320T (es)
RS (2) RS66510B1 (es)
SG (2) SG10202003753PA (es)
SI (2) SI3201320T1 (es)
SM (2) SMT202500074T1 (es)
SV (1) SV2017005416A (es)
TN (1) TN2017000082A1 (es)
TW (4) TW202417620A (es)
WO (1) WO2016054231A1 (es)
ZA (2) ZA201701945B (es)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6851964B2 (ja) 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
IL299470B2 (en) 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
ES3063549T3 (en) * 2015-12-30 2026-04-17 Amicus Therapeutics Inc Augmented acid alpha-glucosidase for the treatment of pompe disease
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
EP4793349A2 (en) * 2016-03-30 2026-08-19 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
EA039750B1 (ru) * 2017-03-30 2022-03-10 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
CN119113091A (zh) * 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
BR112022004000A2 (pt) 2019-09-06 2022-05-31 Amicus Therapeutics Inc Método para captura e purificação de biológicas
JP2024506346A (ja) * 2021-02-11 2024-02-13 アミカス セラピューティックス インコーポレイテッド 組換えヒト酸性アルファグルコシダーゼ及びその使用
AU2022246021A1 (en) * 2021-03-23 2023-10-05 Glycoera Ag Glycan-mediated protein degradation
WO2023150387A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising acid alpha glucosidase and methods of use thereof
WO2023215865A1 (en) 2022-05-05 2023-11-09 Amicus Therapeutics, Inc. Methods for treating pompe disease
WO2024119091A1 (en) 2022-12-02 2024-06-06 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients
AR131256A1 (es) 2022-12-02 2025-03-05 Amicus Therapeutics Inc Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos
WO2026006625A1 (en) 2024-06-26 2026-01-02 Amicus Therapeutics, Inc. Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4837237A (en) 1985-07-09 1989-06-06 Fred Hutchinson Cancer Research Center Therapy using glucosidase processing inhibitors
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
US4985445A (en) 1988-02-12 1991-01-15 Meiji Seika Kaisha, Ltd. Cancer cell metastasis inhibitors and novel compounds
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5179023A (en) 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
US5011829A (en) 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5103008A (en) 1989-08-17 1992-04-07 Monsanto Company Compound, N-butyl-deoxynojirimycin-6-phosphate
US5401650A (en) 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6210666B1 (en) 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
JP2001522833A (ja) 1997-11-10 2001-11-20 ジー・ディー・サール・アンド・カンパニー 多剤耐性を治療するためのアルキル化イミノ糖類の使用
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
DK2020438T3 (en) 1998-12-07 2018-07-23 Genzyme Corp Treatment of Pompe disease
ES2311921T3 (es) 1999-02-12 2009-02-16 United Therapeutics Corporation N-(8,8,8-trifluorooctil)1-5,-didesoxi-1,5-imino-d-glucitol para tratar infecciones por el virus de la hepatitis.
JP2003505430A (ja) 1999-07-26 2003-02-12 ジー・ディー・サール・アンド・カンパニー デオキシノジリマイシンの長鎖ｎ−アルキル誘導体およびグルコセレブロシダーゼ酵素の糖脂質異常蓄積疾患の治療用の薬剤の製造のための使用
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7056712B2 (en) 2000-07-18 2006-06-06 Duke University Treatment of glycogen storage disease type II
CN1638739A (zh) 2000-08-18 2005-07-13 法玛西雅厄普约翰美国公司 治疗成瘾性障碍的化合物
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
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CN1604788B (zh) 2001-10-16 2013-04-17 里克斯金蒂克斯公司 高浓度蛋白制剂及其制备方法
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
JP5589165B2 (ja) 2003-01-31 2014-09-17 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
FR2861991B1 (fr) 2003-11-07 2008-01-18 Centre Nat Rech Scient Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
DE602005020745D1 (de) 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
US20060142234A1 (en) 2004-12-23 2006-06-29 Guohua Chen Injectable non-aqueous suspension
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
AU2007322123A1 (en) 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
CN101636200A (zh) * 2006-11-13 2010-01-27 齐斯特治疗公司 用于治疗庞贝氏症的方法
EP2155197A4 (en) 2007-03-09 2011-10-12 Link Medicine Corp TREATMENT OF LYSOSOMAL STORAGE DISEASES
AU2008245578A1 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20100317690A1 (en) 2007-11-21 2010-12-16 Summit Corporation Plc Treatment of protein folding disorders
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
AU2009223125A1 (en) 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for Pompe disease
JP2011512876A (ja) 2008-03-12 2011-04-28 アミカス セラピューティックス インコーポレイテッド 特異的薬理シャペロンを用いたポンペ病の治療および代理マーカーを用いた治療の監視
WO2009137721A2 (en) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
US20110237538A1 (en) 2008-08-06 2011-09-29 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP3608330B1 (en) * 2008-12-16 2022-11-09 Genzyme Corporation Synthetic intermediates for preparing oligosaccharide-protein conjugates
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
JP5645918B2 (ja) 2009-04-09 2014-12-24 アミカス セラピューティックス インコーポレイテッド リソソーム蓄積障害の予防および／または治療方法
US8785168B2 (en) 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
JP5415170B2 (ja) 2009-07-21 2014-02-12 富士フイルム株式会社 複眼撮像装置
KR102000383B1 (ko) * 2009-09-29 2019-07-15 유니버시테이트 젠트 만노스-1-포스포-6-만노스 결합의 포스포-6-만노스로의 가수분해
US20130196410A1 (en) 2010-03-05 2013-08-01 Alnylam Pharmaceuticals, Inc Compositions and methods for modifying the glycosylation pattern of a polypeptide
BR112013007616B1 (pt) * 2010-09-29 2023-01-24 Oxyrane Uk Limited Métodos para retirar o cap ("uncapping") de porções de manose-1- fosfo-6-manose e desmanosilar n-glicanos fosforilados em uma glicoproteína, para direcionar uma glicoproteína para o interior de uma célula de mamífero, e para converter uma glicoproteína de uma primeira forma que não se liga substancialmente a um receptor de manose-6-fosfato em uma segunda forma que se liga substancialmente a um receptor de manose-6-fosfato em uma célula de mamífero
SG10201605874TA (en) 2011-04-22 2016-09-29 Genzyme Corp Modified acid alpha glucosidase with accelerated processing
LT2714752T (lt) * 2011-05-27 2018-03-12 Amicus Therapeutics, Inc. Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui
WO2013013017A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics
WO2013016249A2 (en) * 2011-07-22 2013-01-31 Insight Photonic Solutions, Inc. System and method of dynamic and adaptive creation of a wavelength-continuous and prescribed wavelength versus time sweep from a laser
WO2013091897A1 (en) 2011-12-22 2013-06-27 Centogene Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
RS61598B1 (sr) 2012-05-03 2021-04-29 Amicus Therapeutics Inc Režimi doziranja za tretman pompeove bolesti
US20150147309A1 (en) 2012-06-06 2015-05-28 Fondazione Telethon Allosteric chaperones and uses thereof
TWI529396B (zh) 2014-07-18 2016-04-11 Mpi Corp Probe card and its transfer circuit board and signal feed structure
JP6851964B2 (ja) 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ

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