MX344587B - Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal. - Google Patents

Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal.

Info

Publication number
MX344587B
MX344587B MX2013008215A MX2013008215A MX344587B MX 344587 B MX344587 B MX 344587B MX 2013008215 A MX2013008215 A MX 2013008215A MX 2013008215 A MX2013008215 A MX 2013008215A MX 344587 B MX344587 B MX 344587B
Authority
MX
Mexico
Prior art keywords
combination therapy
protein deficiency
deficiency disorders
treating protein
protein
Prior art date
Application number
MX2013008215A
Other languages
English (en)
Inventor
Fan Jian-Qiang
Original Assignee
Mount Sinai School Of Medicine Of New York 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 New York Univ filed Critical Mount Sinai School Of Medicine Of New York Univ
Publication of MX344587B publication Critical patent/MX344587B/es

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)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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)

Abstract

La presente invención se refiere a métodos para mejorar la terapia de reemplazo de proteínas al combinar la terapia de reemplazo de proteínas con chaperonas de sitios específicos activos (ASSC) para incrementar la estabilidad y eficiencia de la proteína a administrarse. La solicitud además proporciona composiciones que comprenden la proteína purificada y un ASSC, y métodos de tratamiento por la administración de las composiciones.
MX2013008215A 2003-01-31 2004-02-02 Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal. MX344587B (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX344587B true MX344587B (es) 2016-12-20

Family

ID=32850826

Family Applications (2)

Application Number Title Priority Date Filing Date
MXPA05007822A MXPA05007822A (es) 2003-01-31 2004-02-02 Terapia de combinacion para tratar trastornos de la deficiencia de proteinas.
MX2013008215A MX344587B (es) 2003-01-31 2004-02-02 Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MXPA05007822A MXPA05007822A (es) 2003-01-31 2004-02-02 Terapia de combinacion para tratar trastornos de la deficiencia de proteinas.

Country Status (15)

Country Link
US (4) US20040180419A1 (es)
EP (6) EP2444102B1 (es)
JP (5) JP5589165B2 (es)
CN (4) CN102586205A (es)
BR (1) BRPI0407001A (es)
CA (3) CA2814767C (es)
CY (5) CY1116215T1 (es)
DK (5) DK1589993T3 (es)
ES (6) ES2531478T3 (es)
HU (2) HUE033381T2 (es)
IL (6) IL169562A (es)
MX (2) MXPA05007822A (es)
PT (5) PT2857036T (es)
SI (5) SI2441467T1 (es)
WO (1) WO2004069190A2 (es)

Families Citing this family (58)

* 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
CN102586205A (zh) * 2003-01-31 2012-07-18 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
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
WO2006133446A2 (en) 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
AU2007212021B2 (en) 2006-02-07 2013-06-06 Takeda Pharmaceutical Company Limited Stabilized compositions of proteins having a free thiol moiety
EP2533050B3 (en) 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. 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
CA2657164A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
CA2657238A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic disease
CA2657015A1 (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.
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
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
SI2889043T1 (sl) 2008-12-16 2019-08-30 Genzyme Corporation Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein
KR101755133B1 (ko) 2009-02-23 2017-07-06 유나이티드 세러퓨틱스 코오포레이션 이미노슈가 및 바이러스성 질환을 치료하는 방법
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
JP5805630B2 (ja) * 2009-05-26 2015-11-04 アミカス セラピューティックス インコーポレイテッド 生物製剤の製造および精製を改善するための薬理学的シャペロンの利用
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
CA2765086C (en) * 2009-06-12 2015-12-15 United Therapeutics Corporation Iminosugars and methods of treating bunyaviral and togaviral diseases
IN2012DN00352A (es) * 2009-06-16 2015-08-21 Bikam Pharmaceuticals Inc
NZ713967A (en) 2009-07-28 2017-01-27 Shire Human Genetic Therapies Compositions and methods for treating gaucher disease
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
KR101497194B1 (ko) * 2009-09-04 2015-02-27 유나이티드 세러퓨틱스 코오포레이션 오르토믹소바이러스 감염의 치료 방법
WO2011028781A1 (en) * 2009-09-04 2011-03-10 United Therapeutics Corporation Methods of treating poxviral infections
AR078498A1 (es) * 2009-10-01 2011-11-09 Baylor Res Inst Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry
PL2593131T3 (pl) * 2010-06-25 2020-01-31 Shire Human Genetic Therapies, Inc. Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun
CN104857504A (zh) 2010-06-25 2015-08-26 夏尔人类遗传性治疗公司 芳基硫酸酯酶a cns递送的方法和组合物
CN107088225A (zh) * 2011-03-11 2017-08-25 阿米库斯治疗学公司 治疗法布里病的给药方案
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
US9623090B2 (en) 2012-03-02 2017-04-18 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type III gaucher disease
US9404100B2 (en) 2012-03-07 2016-08-02 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of Pompe disease
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
FI3201320T3 (fi) 2014-09-30 2024-01-08 Amicus Therapeutics Inc Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA3010205A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
DK3397273T3 (da) 2015-12-30 2021-08-09 Amicus Therapeutics Inc Forstærket syre-alfa-glucosidase til behandling af pompes sygdom
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
KR20250050123A (ko) 2016-03-30 2025-04-14 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
CA3031249A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
MX2019011406A (es) 2017-03-27 2019-12-16 Regeneron Pharma Metodo de esterilizacion.
PT3624831T (pt) 2017-05-15 2023-07-04 Amicus Therapeutics Inc Alfa-glucosidase ácida humana recombinante
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
IL272246B2 (en) 2017-07-28 2026-01-01 Applied Therapeutics Inc History 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 an aldose reductase inhibitor for the treatment of galactosemia or prevention of galactosemia-related complications
AR113797A1 (es) 2017-10-26 2020-06-10 Shire Human Genetic Therapies Formulaciones que comprenden glucocerebrosidasa e isofagomina
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
KR20220003529A (ko) 2019-04-01 2022-01-10 어플라이드 테라퓨틱스 인크. 알도스 리덕타제의 억제제
WO2020219874A1 (en) * 2019-04-25 2020-10-29 Shire Human Genetic Therapies, Inc. Isofagomine salts, methods of use and formulations
MX2021013511A (es) * 2019-05-07 2022-02-23 Univ Miami Tratamiento y deteccion de neuropatias heredadas y trastornos asociados.
KR20250051157A (ko) 2019-06-05 2025-04-16 리제너론 파아마슈티컬스, 인크. 정밀 투여량 전달을 위한 디바이스 및 방법

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
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 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
HK1001832A1 (en) 1991-04-25 1998-07-10 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
DE69332105T2 (de) 1992-09-29 2003-03-06 Inhale Therapeutic Systems, San Carlos Pulmonale abgabe von aktiven fragmenten des parathormons
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
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
ES2289194T3 (es) 1993-05-27 2008-02-01 Aventis Pharmaceuticals Inc. Bibliotecas en fase solida codificadas, segregadas topologicamente.
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
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
CA2350919A1 (en) 1998-11-13 2000-05-25 Cyclacel Limited Antennapedia homeodomain helix 3 derived translocation vectors
KR20010101131A (ko) 1998-12-07 2001-11-14 추후기재 폼페병의 치료 방법
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
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation 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
CN102586205A (zh) * 2003-01-31 2012-07-18 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
EP1720405A4 (en) 2004-02-06 2008-08-27 Biomarin Pharm Inc PREPARATION OF STRONG PHOSPHORYLATED LYSOSOMAL ENZYMES AND THEIR USE
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
CA2814774A1 (en) 2004-08-19
DK2332567T3 (en) 2015-07-13
ES2686775T3 (es) 2018-10-19
JP2016053071A (ja) 2016-04-14
DK1589993T3 (en) 2015-03-09
CY1116464T1 (el) 2017-03-15
SI2444102T1 (sl) 2015-08-31
IL214440A0 (en) 2011-09-27
BRPI0407001A (pt) 2006-01-10
DK2441467T3 (en) 2015-08-31
ES2541952T3 (es) 2015-07-28
PT2857036T (pt) 2017-03-13
US9597377B2 (en) 2017-03-21
EP3178486A1 (en) 2017-06-14
US20060153829A1 (en) 2006-07-13
ES2545619T3 (es) 2015-09-14
JP2012102128A (ja) 2012-05-31
IL214440A (en) 2017-03-30
HK1207291A1 (en) 2016-01-29
PT2444102E (pt) 2015-09-17
SI1589993T1 (sl) 2015-05-29
ES2541785T3 (es) 2015-07-24
EP1589993B1 (en) 2014-12-17
WO2004069190A2 (en) 2004-08-19
CY1118849T1 (el) 2018-01-10
HUE033381T2 (en) 2017-11-28
IL214441A0 (en) 2011-09-27
PT1589993E (pt) 2015-03-04
SI2332567T1 (sl) 2015-08-31
HK1168778A1 (en) 2013-01-11
CN1756558A (zh) 2006-04-05
EP1589993A4 (en) 2007-09-26
US20180085437A1 (en) 2018-03-29
CY1116463T1 (el) 2017-03-15
CA2514642C (en) 2015-03-17
JP5875870B2 (ja) 2016-03-02
HUE027113T2 (en) 2016-08-29
JP5589165B2 (ja) 2014-09-17
JP5808675B2 (ja) 2015-11-10
US20040180419A1 (en) 2004-09-16
CN102580067A (zh) 2012-07-18
DK2444102T3 (en) 2015-07-13
CA2814774C (en) 2016-03-22
EP2857036A1 (en) 2015-04-08
EP2441467B1 (en) 2015-05-27
EP2857036B1 (en) 2016-12-21
JP2011078413A (ja) 2011-04-21
EP3178486B1 (en) 2018-06-06
SI2441467T1 (sl) 2015-10-30
IL241315A0 (en) 2015-11-30
IL241315A (en) 2016-12-29
PT2332567E (pt) 2015-09-23
CY1116747T1 (el) 2017-03-15
JP2012107020A (ja) 2012-06-07
CN1756558B (zh) 2012-04-25
CA2514642A1 (en) 2004-08-19
CN102586205A (zh) 2012-07-18
CY1116215T1 (el) 2017-02-08
PT2441467E (pt) 2015-10-12
EP1589993A2 (en) 2005-11-02
EP2332567B1 (en) 2015-04-08
US20070178081A1 (en) 2007-08-02
JP2006516645A (ja) 2006-07-06
HK1169033A1 (en) 2013-01-18
ES2531478T3 (es) 2015-03-16
IL169562A (en) 2016-04-21
CA2814767C (en) 2016-03-15
EP2444102B1 (en) 2015-04-08
IL249389A0 (en) 2017-02-28
IL214439A0 (en) 2011-09-27
ES2619353T3 (es) 2017-06-26
CN102580066A (zh) 2012-07-18
DK2857036T3 (en) 2017-03-13
SI2857036T1 (sl) 2017-05-31
JP6211573B2 (ja) 2017-10-11
EP2444102A1 (en) 2012-04-25
MXPA05007822A (es) 2006-02-10
WO2004069190A3 (en) 2005-06-23
CA2814767A1 (en) 2004-08-19
EP2332567A1 (en) 2011-06-15
HK1158955A1 (en) 2012-07-27
EP2441467A1 (en) 2012-04-18

Similar Documents

Publication Publication Date Title
MXPA05007822A (es) Terapia de combinacion para tratar trastornos de la deficiencia de proteinas.
MXPA05007821A (es) Terapia de combinacion para tratar deficiencias de proteinas.
IL267381A (en) A pharmaceutical preparation containing dextromethorphan and quinidine for the treatment of nervous disorders
MX2007004551A (es) Compuestos tiadiazol y metodos de uso.
MY151032A (en) Treatment of tnf? related disorders
MY172641A (en) High concentration antibody and protein formulations
DE60331537D1 (de) Kombinationen zur behandlung von multiplem myelom
EP1670450B8 (en) Pharmaceutical compositions and method of using levodopa and carbidopa
WO2005000215A3 (en) Methods for treating pain
TW200500355A (en) Methods and compositions using immunomodulatory compounds for treatment and management of cancers and other diseases
MXPA04000222A (es) Combinaciones para el tratamiento de desordenes inflamatorios.
TW200806318A (en) Uses of anti-insulin-like growth factor I receptor antibodies
MX2009002893A (es) Tratamiento de trastornos hepaticos mediante la administracion de conjugados de la proteina asociada al receptor (rap).
SG169224A1 (en) Method for treatment of tumors using nordihydrogualaretic acid derivatives
BRPI0418157A (pt) terapêutica de tumores alogênicos
EA200600921A1 (ru) Способы и композиции с применением талидомида для лечения и менеджмента рака и других заболеваний
MX2009003532A (es) Tratamiento de hiperbilirrubinemia infantil utilizando bajas dosis de estansoporfina.
MY135829A (en) Analeptic and drug combinations
AP2004003177A0 (en) Composition and its therapeutic use
GB0308323D0 (en) Electromagnetic stimulation in patients with osteoporosis
MXPA06007007A (es) Derivados de prolina usados como ingredientes farmaceuticos activos para el tratamiento de tumores.
TW200509922A (en) Methods of therapeutic treatment using amounts of retinoid components