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PT2857036T - Terapia combinada para o tratamento de distúrbios de deficiência proteica - Google Patents

Terapia combinada para o tratamento de distúrbios de deficiência proteica

Info

Publication number
PT2857036T
PT2857036T PT141971978T PT14197197T PT2857036T PT 2857036 T PT2857036 T PT 2857036T PT 141971978 T PT141971978 T PT 141971978T PT 14197197 T PT14197197 T PT 14197197T PT 2857036 T PT2857036 T PT 2857036T
Authority
PT
Portugal
Prior art keywords
combination therapy
protein deficiency
deficiency disorders
treating protein
treating
Prior art date
Application number
PT141971978T
Other languages
English (en)
Inventor
Fan Jian-Qiang
Original Assignee
Mount Sinai School Of Medicine Of N Y Univ
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 N Y Univ filed Critical Mount Sinai School Of Medicine Of N Y Univ
Publication of PT2857036T publication Critical patent/PT2857036T/pt

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • 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
    • 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
    • A61P3/02—Nutrients, e.g. vitamins, minerals
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable 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)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Emergency Medicine (AREA)
  • Obesity (AREA)
  • Hematology (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)
PT141971978T 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica PT2857036T (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
PT2857036T true PT2857036T (pt) 2017-03-13

Family

ID=32850826

Family Applications (5)

Application Number Title Priority Date Filing Date
PT04707422T PT1589993E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT110064953T PT2444102E (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
PT141971978T PT2857036T (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica

Family Applications Before (4)

Application Number Title Priority Date Filing Date
PT04707422T PT1589993E (pt) 2003-01-31 2004-02-02 Terapia combinada para o tratamento de distúrbios de deficiência proteica
PT110064953T PT2444102E (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) EP2444102B1 (pt)
JP (5) JP5589165B2 (pt)
CN (4) CN102580067A (pt)
BR (1) BRPI0407001A (pt)
CA (3) CA2814767C (pt)
CY (5) CY1116215T1 (pt)
DK (5) DK2857036T3 (pt)
ES (6) ES2545619T3 (pt)
HU (2) HUE027113T2 (pt)
IL (6) IL169562A (pt)
MX (2) MXPA05007822A (pt)
PT (5) PT1589993E (pt)
SI (5) SI2332567T1 (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
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
EP2444102B1 (en) 2003-01-31 2015-04-08 The 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
EP2932982B1 (en) * 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
WO2007092829A2 (en) 2006-02-07 2007-08-16 Shire Human Genetic Therapies, Inc. Stabilized compositions of proteins having a free thiol moiety
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
AU2007260812B2 (en) * 2006-06-23 2012-08-16 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase
AU2007277033A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic disease
CA2657164A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
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
US9056101B2 (en) * 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AU2009332930A1 (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
HUE044381T2 (hu) 2008-12-16 2019-10-28 Genzyme Corp Szintetikus intermedierek oligoszacharid-fehérje konjugátumok elõállításához
ES2579628T3 (es) 2009-02-23 2016-08-12 Emergent Virology Llc Iminoazúcares y métodos de tratamiento de enfermedades virales
CA2753194A1 (en) * 2009-02-24 2010-09-02 United Therapeutics Corporation Iminosugars and methods of treating arenaviral infections
ES2585584T3 (es) * 2009-05-26 2016-10-06 Amicus Therapeutics, Inc. Utilización de chaperonas farmacológicas para mejorar la fabricación y la purificación de ß-glucosidasa ácida
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
JP5634510B2 (ja) * 2009-06-12 2014-12-03 ユナイテッド セラピューティクス コーポレイション イミノ糖ならびにブニヤウイルスおよびトガウイルス疾患を治療する方法
IN2012DN00352A (pt) * 2009-06-16 2015-08-21 Bikam Pharmaceuticals Inc
RU2568831C2 (ru) 2009-07-28 2015-11-20 Шайр Хьюман Дженетик Терапиз Композиции и способы для лечения болезни гоше
MX2012002486A (es) * 2009-09-04 2012-09-07 United Therapeutics Corp Metodos para tratar infecciones ortomixovirales.
WO2011028779A1 (en) * 2009-09-04 2011-03-10 United Therapeutics Corporation Iminosugars and methods of treating filoviral diseases
CN102625801B (zh) * 2009-09-04 2015-09-09 联合治疗公司 治疗痘病毒感染的方法
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
PL3626258T3 (pl) * 2010-06-25 2022-01-17 Shire Human Genetic Therapies, Inc. Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun
RS61683B1 (sr) 2010-06-25 2021-05-31 Shire Human Genetic Therapies Isporuka terapijskih agenasa u centralni nervni sistem
SG193379A1 (en) 2011-03-11 2013-10-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
BR112014014808A2 (pt) * 2011-12-22 2018-09-25 Centogene Ip Gmbh combinações de um composto que tem a capacidade de reorganizar uma enzima lisossômica e ambroxol e/ou um derivado de ambroxol, sua utilização, preparação farmacêutica e seu método de preparação
KR102096752B1 (ko) 2012-03-02 2020-04-02 샤이어 휴먼 지네틱 테라피즈 인크. Ｉｉｉ형 고셔병을 치료하기 위한 조성물 및 방법
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
WO2014014938A1 (en) * 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
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
KR20250004926A (ko) 2014-09-30 2025-01-08 아미쿠스 세라퓨틱스, 인코포레이티드 개선된 탄수화물을 가지는 고도로 강력한 산성 알파-글루코시다제
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
CN108472340A (zh) 2015-12-30 2018-08-31 阿米库斯治疗学公司 用于治疗庞贝氏病的增强的酸性α-葡糖苷酶
KR102790256B1 (ko) 2016-03-30 2025-04-04 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
WO2017173060A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
IL313907A (en) 2016-07-19 2024-08-01 Amicus Therapeutics Inc Treatment of Fabry disease in patients experienced with ERT-NAIVE and ERT
US10918754B2 (en) 2017-03-27 2021-02-16 Regeneron Pharmaceuticals, Inc. Sterilisation method
DK3624831T5 (da) 2017-05-15 2024-09-02 Amicus Therapeutics Inc Rekombinant human sur alfa-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
EP3700526A4 (en) 2017-10-26 2021-09-08 Takeda Pharmaceutical Company Limited FORMULATIONS WITH GLUCOCEREBROSIDASE AND ISOFAGOMIN
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
EP3966320A4 (en) * 2019-05-07 2023-10-25 University of Miami TREATMENT AND DETECTION OF HEREDITARY NEUROPATHIES AND ASSOCIATED DISORDERS

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
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a selected therapeutic products
JPH07506252A (ja) 1992-04-24 1995-07-13 エス・アール・アイ・インターナシヨナル 真核細胞内でのイン・ビボ相同配列ターゲッティング
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
NZ257212A (en) 1992-09-29 1996-11-26 Inhale Therapeutic Syst Parathyroid hormone formulations comprising a biologically active n-terminal fragment of the hormone
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US6291657B1 (en) 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives
DK0705279T3 (da) 1993-05-27 2003-06-10 Selectide Corp Topologisk adskilte, kodende fastfase-biblioteker
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
US5916911A (en) 1995-09-20 1999-06-29 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
IL135578A0 (en) * 1997-10-29 2001-05-20 Genzyme Corp 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
AU3113700A (en) 1998-12-07 2000-06-26 Genzyme Corporation 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
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) * 2000-06-19 2002-07-18 David Meeker 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
EP2444102B1 (en) * 2003-01-31 2015-04-08 The Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
EP2932982B1 (en) * 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US9056101B2 (en) 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Also Published As

Publication number Publication date
CY1116463T1 (el) 2017-03-15
EP2441467B1 (en) 2015-05-27
HK1168778A1 (en) 2013-01-11
HK1158955A1 (en) 2012-07-27
CN1756558A (zh) 2006-04-05
JP5589165B2 (ja) 2014-09-17
JP5808675B2 (ja) 2015-11-10
SI1589993T1 (sl) 2015-05-29
EP2332567A1 (en) 2011-06-15
PT2444102E (pt) 2015-09-17
ES2531478T3 (es) 2015-03-16
ES2541785T3 (es) 2015-07-24
HK1169033A1 (en) 2013-01-18
IL241315A0 (en) 2015-11-30
CA2814767A1 (en) 2004-08-19
BRPI0407001A (pt) 2006-01-10
CY1116215T1 (el) 2017-02-08
CY1116747T1 (el) 2017-03-15
CA2814774A1 (en) 2004-08-19
EP2857036A1 (en) 2015-04-08
EP2444102A1 (en) 2012-04-25
ES2545619T3 (es) 2015-09-14
ES2619353T3 (es) 2017-06-26
IL169562A (en) 2016-04-21
PT2332567E (pt) 2015-09-23
WO2004069190A3 (en) 2005-06-23
CA2814774C (en) 2016-03-22
US20060153829A1 (en) 2006-07-13
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JP2012102128A (ja) 2012-05-31
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US20040180419A1 (en) 2004-09-16
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PT1589993E (pt) 2015-03-04
CA2814767C (en) 2016-03-15
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SI2857036T1 (sl) 2017-05-31
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DK2444102T3 (en) 2015-07-13
DK2332567T3 (en) 2015-07-13
US20070178081A1 (en) 2007-08-02
SI2332567T1 (sl) 2015-08-31
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ES2686775T3 (es) 2018-10-19
DK1589993T3 (en) 2015-03-09
HK1207291A1 (en) 2016-01-29
CN102580066A (zh) 2012-07-18
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IL214440A (en) 2017-03-30
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US20180085437A1 (en) 2018-03-29
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PT2441467E (pt) 2015-10-12

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