SI1589993T1 - Kombinirana terapija za zdravljenje motenj pomanjkanja proteina - Google Patents

Kombinirana terapija za zdravljenje motenj pomanjkanja proteina

Info

Publication number
SI1589993T1
SI1589993T1 SI200432226T SI200432226T SI1589993T1 SI 1589993 T1 SI1589993 T1 SI 1589993T1 SI 200432226 T SI200432226 T SI 200432226T SI 200432226 T SI200432226 T SI 200432226T SI 1589993 T1 SI1589993 T1 SI 1589993T1
Authority
SI
Slovenia
Prior art keywords
combination therapy
protein deficiency
deficiency disorders
treating protein
treating
Prior art date
Application number
SI200432226T
Other languages
English (en)
Inventor
Jian-Qiang Fan
Original Assignee
Mount Sinai School Of Medicine Of New York University
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 Mount Sinai School Of Medicine Of New York University filed Critical Mount Sinai School Of Medicine Of New York University
Publication of SI1589993T1 publication Critical patent/SI1589993T1/sl

Links

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)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (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)
  • Nutrition Science (AREA)
  • Vascular Medicine (AREA)
  • Endocrinology (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)
SI200432226T 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina SI1589993T1 (sl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US44413603P 2003-01-31 2003-01-31
PCT/US2004/002973 WO2004069190A2 (en) 2003-01-31 2004-02-02 Combination therapy for treating protein deficiency disorders
EP04707422.4A EP1589993B1 (en) 2003-01-31 2004-02-02 Combination therapy for treating protein deficiency disorders

Publications (1)

Publication Number Publication Date
SI1589993T1 true SI1589993T1 (sl) 2015-05-29

Family

ID=32850826

Family Applications (5)

Application Number Title Priority Date Filing Date
SI200432251T SI2332567T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
SI200432378A SI2857036T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteinov
SI200432253T SI2444102T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
SI200432261T SI2441467T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
SI200432226T SI1589993T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina

Family Applications Before (4)

Application Number Title Priority Date Filing Date
SI200432251T SI2332567T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
SI200432378A SI2857036T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteinov
SI200432253T SI2444102T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
SI200432261T SI2441467T1 (sl) 2003-01-31 2004-02-02 Kombinirana terapija za zdravljenje motenj pomanjkanja proteina

Country Status (16)

Country Link
US (4) US20040180419A1 (sl)
EP (6) EP1589993B1 (sl)
JP (5) JP5589165B2 (sl)
CN (4) CN102580067A (sl)
BR (1) BRPI0407001A (sl)
CA (3) CA2814774C (sl)
CY (5) CY1116215T1 (sl)
DK (5) DK2857036T3 (sl)
ES (6) ES2545619T3 (sl)
HK (4) HK1158955A1 (sl)
HU (2) HUE033381T2 (sl)
IL (6) IL169562A (sl)
MX (2) MX344587B (sl)
PT (5) PT1589993E (sl)
SI (5) SI2332567T1 (sl)
WO (1) WO2004069190A2 (sl)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
CA2814774C (en) * 2003-01-31 2016-03-22 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
EP3782655A1 (en) * 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
KR20080033242A (ko) 2005-06-08 2008-04-16 아미쿠스 세라퓨틱스, 인코포레이티드 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는cns 질환의 치료 방법
AU2007212021B2 (en) 2006-02-07 2013-06-06 Takeda Pharmaceutical Company Limited Stabilized compositions of proteins having a free thiol moiety
CA2652553C (en) 2006-05-16 2020-08-18 Amicus Therapeutics, Inc. Use of 1-deoxygalactonojirimycin or a salt thereof in the treatment of fabry disease
CA2656643C (en) * 2006-06-23 2015-08-25 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase
WO2008013986A2 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
EP2069391A4 (en) * 2006-07-27 2009-12-30 Univ Florida COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING EYE DRYING
WO2008013983A2 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Opsin stabilizing compounds and methods of use
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.
WO2009100037A1 (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
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
US8835614B2 (en) 2008-12-16 2014-09-16 Genzyme Corporation Oligosaccharide-protein conjugates
KR20120042716A (ko) 2009-02-23 2012-05-03 유나이티드 세러퓨틱스 코오포레이션 이미노슈가 및 바이러스성 질환을 치료하는 방법
ES2562635T3 (es) * 2009-02-24 2016-03-07 United Therapeutics Corporation Iminoazúcares y métodos de tratamiento de infecciones producidas por arenavirus
AU2010254092B2 (en) * 2009-05-26 2015-11-12 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
KR101463661B1 (ko) 2009-06-12 2014-11-19 유나이티드 세러퓨틱스 코오포레이션 면역당 및 분야바이러스 및 토가바이러스 질환의 치료 방법
IN2012DN00352A (sl) * 2009-06-16 2015-08-21 Bikam Pharmaceuticals Inc
MX2012001268A (es) 2009-07-28 2012-09-12 Shire Human Genetic Therapies Composiciones y metodos para tratar enfermedad de gaucher.
KR101459530B1 (ko) * 2009-09-04 2014-11-07 유나이티드 세러퓨틱스 코오포레이션 폭스바이러스 감염의 치료 방법
US20110065752A1 (en) * 2009-09-04 2011-03-17 United Therapeutics Corporation Methods of treating orthomyxoviral infections
CA2772813A1 (en) * 2009-09-04 2011-03-10 The Chancellor, Masters And Scholars Of The University Of Oxford Iminosugars and methods of treating filoviral diseases
US20110112041A1 (en) * 2009-10-01 2011-05-12 Baylor Research Institute Treatment of Male-Pattern Baldness by Local Induction of the Metabolic Defect of Fabry Disease
PT2593131T (pt) 2010-06-25 2019-10-30 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
KR102151889B1 (ko) * 2010-06-25 2020-09-04 샤이어 휴먼 지네틱 테라피즈 인크. 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
CA2860085A1 (en) * 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
US9623090B2 (en) 2012-03-02 2017-04-18 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
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
EP2874648A4 (en) 2012-07-17 2015-12-30 Amicus Therapeutics Inc CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN
WO2015009596A2 (en) * 2013-07-15 2015-01-22 New Yor University Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases
CN107075468B (zh) 2014-09-30 2021-12-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2017117407A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
RS64843B1 (sr) 2015-12-30 2023-12-29 Amicus Therapeutics Inc Augmentirana kisela alfa-glukozidaza za lečenje pompeove bolesti
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
IL295551A (en) 2016-03-30 2022-10-01 Amicus Therapeutics Inc A method for selecting recombinant proteins with a high amount of mannose-6-phosphate
IL311121A (en) 2017-03-27 2024-04-01 Regeneron Pharma Sterilization method
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
US11590131B2 (en) 2017-07-28 2023-02-28 Applied Therapeutics, Inc. Compositions and methods for treating galactosemia
EP3700526A4 (en) * 2017-10-26 2021-09-08 Takeda Pharmaceutical Company Limited FORMULATIONS CONTAINING GLUCOCEREBROSIDASE AND ISOFAGOMINE
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
CN113906012A (zh) * 2019-04-25 2022-01-07 武田药品工业有限公司 异法戈明盐、使用方法和调配物
MX2021013511A (es) * 2019-05-07 2022-02-23 Univ Miami Tratamiento y deteccion de neuropatias heredadas y trastornos asociados.

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
JP4215273B2 (ja) 1991-04-25 2009-01-28 ブラウン ユニヴァーシティ リサーチ ファンデーション 選択された治療物質放出用の移植可能で生体適合性の免疫遮断性ビークル
CA2118513A1 (en) 1992-04-24 1993-11-11 David A. Zarling In vivo homologous sequence targeting in eukaryotic cells
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
AU688283B2 (en) 1992-09-29 1998-03-12 Novartis Ag Pulmonary delivery of active fragments of parathyroid hormone
US5250703A (en) * 1992-09-29 1993-10-05 Monsanto Company Pyrrolidin-3,4-diol derivatives of heptitols and preparation thereof
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
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
WO1994028028A1 (en) 1993-05-27 1994-12-08 Selectide Corporation 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
WO2000009153A1 (en) * 1997-10-29 2000-02-24 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
JP2002530059A (ja) 1998-11-13 2002-09-17 サイクラセル・リミテッド 輸送ベクター
WO2000034451A1 (en) 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Treatment of pompe's disease
AU6050600A (en) * 1999-07-26 2001-02-13 G.D. Searle & Co. Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidaseenzyme for the manufacture of medicament for the treatment of glycolipid storag e diseases
US6188045B1 (en) 2000-04-03 2001-02-13 Alto-Shaam, Inc. Combination oven with three-stage water atomizer
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
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
WO2004037373A2 (en) 2002-10-21 2004-05-06 The Scripps Research Institute CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE
CA2814774C (en) 2003-01-31 2016-03-22 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
AU2005211775B2 (en) 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
EP3782655A1 (en) * 2005-05-17 2021-02-24 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
DK2441467T3 (en) 2015-08-31
ES2545619T3 (es) 2015-09-14
CA2814774C (en) 2016-03-22
PT2857036T (pt) 2017-03-13
PT2441467E (pt) 2015-10-12
JP5875870B2 (ja) 2016-03-02
ES2531478T3 (es) 2015-03-16
DK2332567T3 (en) 2015-07-13
CN1756558A (zh) 2006-04-05
CY1116464T1 (el) 2017-03-15
DK1589993T3 (en) 2015-03-09
JP5589165B2 (ja) 2014-09-17
JP5808675B2 (ja) 2015-11-10
WO2004069190A2 (en) 2004-08-19
SI2332567T1 (sl) 2015-08-31
HK1169033A1 (en) 2013-01-18
EP2441467A1 (en) 2012-04-18
US9597377B2 (en) 2017-03-21
EP2444102B1 (en) 2015-04-08
JP2012102128A (ja) 2012-05-31
ES2541785T3 (es) 2015-07-24
HK1207291A1 (en) 2016-01-29
DK2444102T3 (en) 2015-07-13
ES2619353T3 (es) 2017-06-26
US20180085437A1 (en) 2018-03-29
JP2012107020A (ja) 2012-06-07
EP2857036A1 (en) 2015-04-08
CA2814774A1 (en) 2004-08-19
EP3178486B1 (en) 2018-06-06
SI2444102T1 (sl) 2015-08-31
EP2857036B1 (en) 2016-12-21
SI2857036T1 (sl) 2017-05-31
CN102580066A (zh) 2012-07-18
CY1116463T1 (el) 2017-03-15
CA2514642C (en) 2015-03-17
CN102586205A (zh) 2012-07-18
PT2444102E (pt) 2015-09-17
US20040180419A1 (en) 2004-09-16
US20070178081A1 (en) 2007-08-02
CY1118849T1 (el) 2018-01-10
HUE033381T2 (en) 2017-11-28
CN1756558B (zh) 2012-04-25
JP2016053071A (ja) 2016-04-14
CY1116215T1 (el) 2017-02-08
EP2444102A1 (en) 2012-04-25
EP1589993B1 (en) 2014-12-17
IL169562A (en) 2016-04-21
HK1168778A1 (en) 2013-01-11
JP2006516645A (ja) 2006-07-06
WO2004069190A3 (en) 2005-06-23
IL241315A (en) 2016-12-29
EP3178486A1 (en) 2017-06-14
JP2011078413A (ja) 2011-04-21
PT2332567E (pt) 2015-09-23
IL214441A0 (en) 2011-09-27
CA2814767C (en) 2016-03-15
MX344587B (es) 2016-12-20
PT1589993E (pt) 2015-03-04
IL214440A (en) 2017-03-30
HUE027113T2 (en) 2016-08-29
ES2686775T3 (es) 2018-10-19
CA2814767A1 (en) 2004-08-19
MXPA05007822A (es) 2006-02-10
CY1116747T1 (el) 2017-03-15
US20060153829A1 (en) 2006-07-13
ES2541952T3 (es) 2015-07-28
BRPI0407001A (pt) 2006-01-10
EP2441467B1 (en) 2015-05-27
CA2514642A1 (en) 2004-08-19
EP2332567B1 (en) 2015-04-08
SI2441467T1 (sl) 2015-10-30
EP1589993A4 (en) 2007-09-26
IL241315A0 (en) 2015-11-30
HK1158955A1 (en) 2012-07-27
JP6211573B2 (ja) 2017-10-11
CN102580067A (zh) 2012-07-18
EP2332567A1 (en) 2011-06-15
DK2857036T3 (en) 2017-03-13
EP1589993A2 (en) 2005-11-02
IL249389A0 (en) 2017-02-28
IL214439A0 (en) 2011-09-27
IL214440A0 (en) 2011-09-27

Similar Documents

Publication Publication Date Title
IL249389A0 (en) Combined therapy for the treatment of disorders associated with protein deficiency
IL169633A0 (en) Combination therapy for treating protein deficiencies
IL175055A0 (en) Combinations useful for the treatment of neuronal disorders
ZA200508427B (en) Methods for treating interleuking-6 related diseases
HK1092367A1 (en) Combination therapy for the treatment of ocular neovascular disorders
GB0201850D0 (en) Therapeutic treatment
GB0316912D0 (en) Therapeutic treatment
PL378108A1 (pl) Leczenie łączone do terapii zapalnych zaburzeń immunologicznych
GB0320806D0 (en) Therapeutic treatment
IL172249A0 (en) Methods for treating protein aggregation disorders
IL162818A0 (en) Combination therapy for the treatment of bacterialinfections
EP1646381A4 (en) COMBINED THERAPY FOR THE TREATMENT OF CHRONIC INFLAMMATORY DISEASES
GB0329275D0 (en) Therapeutic treatment
GB0307863D0 (en) Therapeutic treatment
ZA200505996B (en) Combination therapy for the treatment of immunoinflammatory disorders
GB0313772D0 (en) Therapeutic treatment
GB2397018B (en) Combination therapy for treating disease
GB0217271D0 (en) Therapeutic treatment for tinnitus
GB0321234D0 (en) Therapeutic treatment
GB0321235D0 (en) Therapeutic treatment
GB0322000D0 (en) Therapeutic treatment
GB0321649D0 (en) Therapeutic treatment
GB0321236D0 (en) Therapeutic treatment
GB0300881D0 (en) Therapeutic treatment
GB0321650D0 (en) Therapeutic treatment