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PT1589993E - Terapia combinada para o tratamento de distúrbios de deficiência proteica - Google Patents

Terapia combinada para o tratamento de distúrbios de deficiência proteica Download PDF

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Publication number
PT1589993E
PT1589993E PT04707422T PT04707422T PT1589993E PT 1589993 E PT1589993 E PT 1589993E PT 04707422 T PT04707422 T PT 04707422T PT 04707422 T PT04707422 T PT 04707422T PT 1589993 E PT1589993 E PT 1589993E
Authority
PT
Portugal
Prior art keywords
combination therapy
protein deficiency
deficiency disorders
treating protein
treating
Prior art date
Application number
PT04707422T
Other languages
English (en)
Inventor
Jian-Qiang Fan
Original Assignee
Sinai School Medicine
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 Sinai School Medicine filed Critical Sinai School Medicine
Publication of PT1589993E publication Critical patent/PT1589993E/pt

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/468-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/02Nutrients, e.g. vitamins, minerals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/14Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Endocrinology (AREA)
  • Nutrition Science (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
PT04707422T 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica PT1589993E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US44413603P 2003-01-31 2003-01-31

Publications (1)

Publication Number Publication Date
PT1589993E true PT1589993E (pt) 2015-03-04

Family

ID=32850826

Family Applications (5)

Application Number Title Priority Date Filing Date
PT110064953T PT2444102E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT04707422T PT1589993E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT141971978T PT2857036T (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT100115989T PT2332567E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT110065042T PT2441467E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT110064953T PT2444102E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica

Family Applications After (3)

Application Number Title Priority Date Filing Date
PT141971978T PT2857036T (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT100115989T PT2332567E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT110065042T PT2441467E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de transtornos associados a uma deficiência proteica

Country Status (15)

Country Link
US (4) US20040180419A1 (pt)
EP (6) EP3178486B1 (pt)
JP (5) JP5589165B2 (pt)
CN (4) CN102580067A (pt)
BR (1) BRPI0407001A (pt)
CA (3) CA2814767C (pt)
CY (5) CY1116215T1 (pt)
DK (5) DK1589993T3 (pt)
ES (6) ES2541785T3 (pt)
HU (2) HUE033381T2 (pt)
IL (6) IL169562A (pt)
MX (2) MX344587B (pt)
PT (5) PT2444102E (pt)
SI (5) SI2444102T1 (pt)
WO (1) WO2004069190A2 (pt)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation 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
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
FR2861991B1 (fr) 2003-11-07 2008-01-18 Centre Nat Rech Scient Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
US20050256168A1 (en) * 2004-04-28 2005-11-17 Block Timothy M Compositions for oral administration for the treatment of interferon-responsive disorders
EP3441090A1 (en) * 2005-05-17 2019-02-13 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
MX2007015602A (es) 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
SI1986612T1 (sl) 2006-02-07 2013-01-31 Shire Human Genetic Therapies, Inc. Stabilizirana spojina glukocerebrosidaze
EP2024745B1 (en) 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
JP5303458B2 (ja) 2006-06-23 2013-10-02 アミークス セラピューティックス インコーポレイテッド β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
WO2008013986A2 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
AU2007277032A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Opsin stabilizing compounds and methods of use
US20100104644A1 (en) * 2006-07-27 2010-04-29 University Of Florida Research Foundation, Inc. Compositions and Methods for Treating or Preventing Ophthalmic Disease
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
CA2712593A1 (en) * 2008-02-01 2009-08-13 The Scripps Research Institute Methods for treating a condition characterized by dysfunction in protein homeostasis
WO2010048532A1 (en) * 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP2465542B1 (en) 2008-12-16 2015-01-21 Genzyme Corporation Oligosaccharide-protein conjugates
ES2579628T3 (es) * 2009-02-23 2016-08-12 Emergent Virology Llc Iminoazúcares y métodos de tratamiento de enfermedades virales
CN102625660B (zh) * 2009-02-24 2015-03-11 联合治疗公司 亚氨基糖以及治疗沙粒病毒感染的方法
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
US8426445B2 (en) * 2009-06-12 2013-04-23 United Therapeutics Corporation Iminosugars and methods of treating bunyaviral and togaviral diseases
IN2012DN00352A (pt) * 2009-06-16 2015-08-21 Bikam Pharmaceuticals Inc
NZ598156A (en) 2009-07-28 2014-06-27 Shire Human Genetic Therapies Compositions and methods for treating gaucher disease
WO2011028781A1 (en) * 2009-09-04 2011-03-10 United Therapeutics Corporation Methods of treating poxviral infections
US20110065752A1 (en) * 2009-09-04 2011-03-17 United Therapeutics Corporation Methods of treating orthomyxoviral infections
CN105748476A (zh) * 2009-09-04 2016-07-13 联合治疗公司 亚氨基糖以及治疗丝状病毒性疾病的方法
WO2011041718A2 (en) * 2009-10-01 2011-04-07 Baylor Research Institute Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
PT2588130T (pt) 2010-06-25 2016-11-25 Shire Human Genetic Therapies Entrega de agentes terapêuticos no cns
UA115649C2 (uk) * 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Способи та композиції для доставки до цнс ідуронат-2-сульфатази
EA031874B1 (ru) 2011-03-11 2019-03-29 Амикус Терапьютикс, Инк. Способ лечения болезни фабри
AU2012359165A1 (en) * 2011-12-22 2014-07-10 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
CA2865614A1 (en) 2012-03-02 2013-09-06 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type iii gaucher disease
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
WO2014014938A1 (en) * 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
US20160346363A1 (en) * 2013-07-15 2016-12-01 New York University Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases
MY186336A (en) 2014-09-30 2021-07-13 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
PL3957320T3 (pl) 2015-12-30 2024-02-19 Amicus Therapeutics, Inc. Wzmocniona kwaśna alfa-glukozydaza do leczenia choroby Pompego
CA3010205A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
IL301319A (en) 2016-03-30 2023-05-01 Amicus Therapeutics Inc Formulations that include recombinant acid alpha-glucosidase
TWI875676B (zh) 2016-07-19 2025-03-11 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症
IL315639A (en) 2017-03-27 2024-11-01 Regeneron Pharma Sterilization method
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
JP7066157B2 (ja) 2017-06-14 2022-05-13 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
IL272246B1 (en) 2017-07-28 2025-09-01 Applied Therapeutics Inc Derivatives of 2-(4-oxo/thioketone/azo-3-((substituted)benzo[d]thiazol-2-yl)methyl)- 3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid for use as aldose reductase inhibitors in treating galactosemia or preventing complications associated with galactosemia
KR20240171186A (ko) 2017-10-26 2024-12-06 다케다 야쿠힌 고교 가부시키가이샤 글루코세레브로시다제 및 이소파고민을 포함하는 제제
NL2021840B1 (en) * 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
WO2020046132A1 (en) * 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
US12286400B2 (en) 2019-04-25 2025-04-29 Takeda Pharmaceutical Company Limited Isofagomine salts, methods of use and formulations
CN113966396A (zh) * 2019-05-07 2022-01-21 迈阿密大学 遗传性神经病和相关障碍的治疗和检测

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3607858A (en) * 1970-03-31 1971-09-21 American Cyanamid Co Process for preparing lyophilized human blood proteins such as gamma globulin in the presence of a nonionic surfactant
US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
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
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
US5030638A (en) * 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US5139749A (en) 1990-06-22 1992-08-18 Tas, Inc. Fluidized calcining process
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
EP0585368B1 (en) 1991-04-25 1997-08-06 Brown University Research Foundation Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products
DE69333955D1 (de) 1992-04-24 2006-02-02 Stanford Res Inst Int Targeting homologer sequenzen in eukaryotenzellen
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
US5250703A (en) * 1992-09-29 1993-10-05 Monsanto Company Pyrrolidin-3,4-diol derivatives of heptitols and preparation thereof
DK0679088T3 (da) 1992-09-29 2002-10-28 Inhale Therapeutic Syst Pulmonal levering af aktive fragmenter af parathyreoideaea hormon
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US6291657B1 (en) 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives
AU686186B2 (en) 1993-05-27 1998-02-05 Aventisub Ii Inc. Topologically segregated, encoded solid phase libraries
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
US20020013953A1 (en) 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
WO1997010817A1 (en) 1995-09-20 1997-03-27 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
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
US5948653A (en) 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US6210666B1 (en) 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
EP2147681A1 (en) * 1997-10-29 2010-01-27 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
CA2350919A1 (en) 1998-11-13 2000-05-25 Cyclacel Limited Antennapedia homeodomain helix 3 derived translocation vectors
AU3113700A (en) 1998-12-07 2000-06-26 Genzyme Corporation Treatment of pompe's disease
EP1196190B1 (en) 1999-07-26 2003-03-19 G.D. SEARLE & CO. Use of long-chain n-alkyl derivatives of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of a medicament for the treatment of glycolipid storage diseases
US6188045B1 (en) 2000-04-03 2001-02-13 Alto-Shaam, Inc. Combination oven with three-stage water atomizer
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation 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
US20020102329A1 (en) * 2000-07-22 2002-08-01 Michael Lanahan Methods for high-temperature hydrolysis of galactose-containing oligosaccharides in complex mixtures
US7001994B2 (en) * 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
AU2003284886A1 (en) 2002-10-21 2004-05-13 William E. Balch CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
CA2556245A1 (en) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
EP3441090A1 (en) * 2005-05-17 2019-02-13 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
MX2009011473A (es) 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.

Also Published As

Publication number Publication date
CY1116464T1 (el) 2017-03-15
SI2441467T1 (sl) 2015-10-30
ES2619353T3 (es) 2017-06-26
DK2857036T3 (en) 2017-03-13
US9597377B2 (en) 2017-03-21
HK1207291A1 (en) 2016-01-29
CY1116215T1 (el) 2017-02-08
PT2441467E (pt) 2015-10-12
CA2814774C (en) 2016-03-22
US20040180419A1 (en) 2004-09-16
SI2857036T1 (sl) 2017-05-31
CA2814767A1 (en) 2004-08-19
CA2514642C (en) 2015-03-17
MX344587B (es) 2016-12-20
EP2444102A1 (en) 2012-04-25
JP2012102128A (ja) 2012-05-31
JP2016053071A (ja) 2016-04-14
EP2332567B1 (en) 2015-04-08
ES2531478T3 (es) 2015-03-16
CA2814774A1 (en) 2004-08-19
PT2332567E (pt) 2015-09-23
ES2541952T3 (es) 2015-07-28
EP2857036A1 (en) 2015-04-08
DK2444102T3 (en) 2015-07-13
CN102580067A (zh) 2012-07-18
CY1116747T1 (el) 2017-03-15
EP2441467B1 (en) 2015-05-27
EP1589993A4 (en) 2007-09-26
DK2332567T3 (en) 2015-07-13
IL241315A (en) 2016-12-29
EP2441467A1 (en) 2012-04-18
BRPI0407001A (pt) 2006-01-10
EP1589993B1 (en) 2014-12-17
US20180085437A1 (en) 2018-03-29
CN1756558B (zh) 2012-04-25
IL169562A (en) 2016-04-21
MXPA05007822A (es) 2006-02-10
DK2441467T3 (en) 2015-08-31
WO2004069190A3 (en) 2005-06-23
IL214440A0 (en) 2011-09-27
EP2857036B1 (en) 2016-12-21
PT2444102E (pt) 2015-09-17
EP3178486A1 (en) 2017-06-14
IL214439A0 (en) 2011-09-27
SI2332567T1 (sl) 2015-08-31
IL249389A0 (en) 2017-02-28
HK1168778A1 (en) 2013-01-11
HK1158955A1 (en) 2012-07-27
CY1116463T1 (el) 2017-03-15
CN102586205A (zh) 2012-07-18
CA2814767C (en) 2016-03-15
EP2332567A1 (en) 2011-06-15
IL241315A0 (en) 2015-11-30
IL214441A0 (en) 2011-09-27
HK1169033A1 (en) 2013-01-18
JP5589165B2 (ja) 2014-09-17
JP5808675B2 (ja) 2015-11-10
ES2541785T3 (es) 2015-07-24
JP2006516645A (ja) 2006-07-06
ES2686775T3 (es) 2018-10-19
JP5875870B2 (ja) 2016-03-02
DK1589993T3 (en) 2015-03-09
WO2004069190A2 (en) 2004-08-19
SI1589993T1 (sl) 2015-05-29
US20060153829A1 (en) 2006-07-13
CN102580066A (zh) 2012-07-18
IL214440A (en) 2017-03-30
JP2012107020A (ja) 2012-06-07
CN1756558A (zh) 2006-04-05
EP3178486B1 (en) 2018-06-06
CY1118849T1 (el) 2018-01-10
EP1589993A2 (en) 2005-11-02
CA2514642A1 (en) 2004-08-19
HUE033381T2 (en) 2017-11-28
ES2545619T3 (es) 2015-09-14
HUE027113T2 (en) 2016-08-29
EP2444102B1 (en) 2015-04-08
JP6211573B2 (ja) 2017-10-11
PT2857036T (pt) 2017-03-13
US20070178081A1 (en) 2007-08-02
SI2444102T1 (sl) 2015-08-31
JP2011078413A (ja) 2011-04-21

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