CY1116747T1 - Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης - Google Patents

Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Info

Publication number
CY1116747T1
CY1116747T1 CY20151100746T CY151100746T CY1116747T1 CY 1116747 T1 CY1116747 T1 CY 1116747T1 CY 20151100746 T CY20151100746 T CY 20151100746T CY 151100746 T CY151100746 T CY 151100746T CY 1116747 T1 CY1116747 T1 CY 1116747T1
Authority
CY
Cyprus
Prior art keywords
protein
combination therapy
therapeutic treatment
protein deficiency
deficiency disorders
Prior art date
Application number
CY20151100746T
Other languages
English (en)
Inventor
Jian-Qiang Fan
Original Assignee
The 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 The Mount Sinai School Of Medicine Of New York University filed Critical The Mount Sinai School Of Medicine Of New York University
Publication of CY1116747T1 publication Critical patent/CY1116747T1/el

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, ***e
    • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Endocrinology (AREA)
  • Nutrition Science (AREA)
  • Vascular Medicine (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

Η παρούσα αίτηση παρέχει μεθόδους βελτίωσης θεραπείας υποκατάστασης πρωτεΐνης μέσω συνδυασμού θεραπείας υποκατάστασης πρωτεΐνης με ενεργές τοπο-ειδικές χαπερόνες (ASSC), ώστε να αυξηθεί η σταθερότητα και η αποτελεσματικότητα της εκάστοτε χορηγούμενης πρωτεΐνης. Η αίτηση περαιτέρω παρέχει συνθέσεις που περιλαμβάνουν την καθαρισμένη πρωτεΐνη και μία ASSC, και μεθόδους θεραπευτικής αγωγής μέσω χορήγησης των συνθέσεων.
CY20151100746T 2003-01-31 2015-08-26 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης CY1116747T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44413603P 2003-01-31 2003-01-31
EP11006504.2A EP2441467B1 (en) 2003-01-31 2004-02-02 Combination therapy for treating protein deficiency disorders

Publications (1)

Publication Number Publication Date
CY1116747T1 true CY1116747T1 (el) 2017-03-15

Family

ID=32850826

Family Applications (5)

Application Number Title Priority Date Filing Date
CY20151100242T CY1116215T1 (el) 2003-01-31 2015-03-11 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20151100594T CY1116464T1 (el) 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20151100593T CY1116463T1 (el) 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20151100746T CY1116747T1 (el) 2003-01-31 2015-08-26 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20171100345T CY1118849T1 (el) 2003-01-31 2017-03-20 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Family Applications Before (3)

Application Number Title Priority Date Filing Date
CY20151100242T CY1116215T1 (el) 2003-01-31 2015-03-11 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20151100594T CY1116464T1 (el) 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY20151100593T CY1116463T1 (el) 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Family Applications After (1)

Application Number Title Priority Date Filing Date
CY20171100345T CY1118849T1 (el) 2003-01-31 2017-03-20 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Country Status (16)

Country Link
US (4) US20040180419A1 (el)
EP (6) EP2857036B1 (el)
JP (5) JP5589165B2 (el)
CN (4) CN102580067A (el)
BR (1) BRPI0407001A (el)
CA (3) CA2814774C (el)
CY (5) CY1116215T1 (el)
DK (5) DK2441467T3 (el)
ES (6) ES2619353T3 (el)
HK (4) HK1158955A1 (el)
HU (2) HUE027113T2 (el)
IL (6) IL169562A (el)
MX (2) MXPA05007822A (el)
PT (5) PT1589993E (el)
SI (5) SI2857036T1 (el)
WO (1) WO2004069190A2 (el)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
ES2619353T3 (es) * 2003-01-31 2017-06-26 Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
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
ES2572148T3 (es) 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
CA2611011C (en) 2005-06-08 2014-01-28 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
ES2391657T3 (es) 2006-02-07 2012-11-28 Shire Human Genetic Therapies, Inc. Composiciones estabilizadas de proteínas que tienen un resto tiol libre
EP2955520B1 (en) 2006-05-16 2018-10-31 Amicus Therapeutics, Inc. Treatment options for fabry disease
US7829579B2 (en) 2006-06-23 2010-11-09 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase
AU2007277032A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Opsin stabilizing compounds and methods of use
CA2657164A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
WO2008013984A2 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic disease
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20090203605A1 (en) * 2008-02-01 2009-08-13 The Scripps Research Institute Methods For Treating A Condition Characterized By Dysfunction In Protein Homeostasis
US8321148B2 (en) * 2008-10-24 2012-11-27 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
ES2579628T3 (es) 2009-02-23 2016-08-12 Emergent Virology Llc Iminoazúcares y métodos de tratamiento de enfermedades virales
JP5951996B2 (ja) * 2009-02-24 2016-07-13 ユナイテッド セラピューティクス コーポレーション イミノ糖及びアレナウイルス感染症を治療する方法
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V 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
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 (el) * 2009-06-16 2015-08-21 Bikam Pharmaceuticals Inc
RU2733466C2 (ru) 2009-07-28 2020-10-01 Шайр Хьюман Дженетик Терапиз Композиции и способы для лечения болезни гоше
ES2527623T3 (es) * 2009-09-04 2015-01-27 United Therapeutics Corporation Iminoazúcares para su uso en el tratamiento de enfermedades por filovirus
CN102625796B (zh) * 2009-09-04 2015-03-11 联合治疗公司 治疗正粘病毒感染的方法
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
DK3103469T3 (en) 2010-06-25 2021-02-22 Shire Human Genetic Therapies Indgivelse af terapeutiske midler til centralnervesystemet
JP6045492B2 (ja) * 2010-06-25 2016-12-14 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物
US20140219986A1 (en) * 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry disease
ES2864774T3 (es) * 2011-12-22 2021-10-14 Centogene Ip Gmbh Combinación de un compuesto que tiene la capacidad de reorganizar una enzima lisosomal y Ambroxol y/o un derivado de Ambroxol
KR20140138850A (ko) 2012-03-02 2014-12-04 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
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
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
PT3957320T (pt) 2015-12-30 2023-11-23 Amicus Therapeutics Inc Alfa-glicosidase ácida aumentada para o tratamento da doença de pompe
KR20220145918A (ko) 2016-03-30 2022-10-31 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US10918754B2 (en) 2017-03-27 2021-02-16 Regeneron Pharmaceuticals, Inc. Sterilisation 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
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
WO2020046132A1 (en) * 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
NL2021840B1 (en) * 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
WO2020219874A1 (en) * 2019-04-25 2020-10-29 Shire Human Genetic Therapies, Inc. 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
CA2109085C (en) 1991-04-25 2003-03-11 Keith E. Dionne Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products
AU4115693A (en) * 1992-04-24 1993-11-29 Sri International 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
US5250703A (en) * 1992-09-29 1993-10-05 Monsanto Company Pyrrolidin-3,4-diol derivatives of heptitols and preparation thereof
BR9307141A (pt) 1992-09-29 1999-03-30 Inhale Therapeutic Syst Processo para a liberação sistência pulsátil de um fragmento ativo de hormônio de paratiróide (PTH) para um hospedeiro mamífero e para um paciente e composição farmacêutica
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
ES2204921T3 (es) 1993-05-27 2004-05-01 Selectide Corporation Bibliotecas de 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
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
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
ATE419273T1 (de) 1998-11-13 2009-01-15 Cyclacel Ltd Translokationsvektoren abgeleitet von der antennapedia homeodomain helix 3
KR20010101131A (ko) 1998-12-07 2001-11-14 추후기재 폼페병의 치료 방법
ATE234626T1 (de) * 1999-07-26 2003-04-15 Searle & Co Verwendung von langkettigen n-alkyl derivaten von deoxynojirimycin mit glucocerebrosidase enzyme zur herstellung eines medikaments zur behandlung von mit glykolipiden akkumulation zusammenhängenden krankheiten
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
ES2619353T3 (es) 2003-01-31 2017-06-26 Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
EP1720405A4 (en) 2004-02-06 2008-08-27 Biomarin Pharm Inc MANUFACTURE OF HIGHLY PHOSPHORYLATED LYSOSOMAL ENZYMES AND USES THEREOF
ES2572148T3 (es) * 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Also Published As

Publication number Publication date
JP2012107020A (ja) 2012-06-07
EP2441467A1 (en) 2012-04-18
HK1168778A1 (en) 2013-01-11
EP2332567B1 (en) 2015-04-08
BRPI0407001A (pt) 2006-01-10
DK1589993T3 (en) 2015-03-09
JP5875870B2 (ja) 2016-03-02
US20180085437A1 (en) 2018-03-29
CA2814767C (en) 2016-03-15
EP1589993B1 (en) 2014-12-17
CA2514642C (en) 2015-03-17
DK2444102T3 (en) 2015-07-13
EP2441467B1 (en) 2015-05-27
PT2441467E (pt) 2015-10-12
JP6211573B2 (ja) 2017-10-11
CA2814774C (en) 2016-03-22
CY1118849T1 (el) 2018-01-10
HUE033381T2 (en) 2017-11-28
EP3178486B1 (en) 2018-06-06
WO2004069190A3 (en) 2005-06-23
CN1756558A (zh) 2006-04-05
SI2857036T1 (sl) 2017-05-31
JP2016053071A (ja) 2016-04-14
IL214441A0 (en) 2011-09-27
JP2011078413A (ja) 2011-04-21
SI2444102T1 (sl) 2015-08-31
HK1158955A1 (en) 2012-07-27
PT2444102E (pt) 2015-09-17
ES2531478T3 (es) 2015-03-16
PT1589993E (pt) 2015-03-04
PT2332567E (pt) 2015-09-23
ES2686775T3 (es) 2018-10-19
IL214440A0 (en) 2011-09-27
DK2441467T3 (en) 2015-08-31
EP2332567A1 (en) 2011-06-15
CN102580066A (zh) 2012-07-18
SI2332567T1 (sl) 2015-08-31
CN102586205A (zh) 2012-07-18
HUE027113T2 (en) 2016-08-29
EP3178486A1 (en) 2017-06-14
US20070178081A1 (en) 2007-08-02
DK2332567T3 (en) 2015-07-13
CA2514642A1 (en) 2004-08-19
ES2619353T3 (es) 2017-06-26
US9597377B2 (en) 2017-03-21
JP5589165B2 (ja) 2014-09-17
JP5808675B2 (ja) 2015-11-10
CY1116463T1 (el) 2017-03-15
SI2441467T1 (sl) 2015-10-30
IL214439A0 (en) 2011-09-27
CA2814774A1 (en) 2004-08-19
CN1756558B (zh) 2012-04-25
PT2857036T (pt) 2017-03-13
ES2545619T3 (es) 2015-09-14
US20060153829A1 (en) 2006-07-13
MX344587B (es) 2016-12-20
HK1207291A1 (en) 2016-01-29
DK2857036T3 (en) 2017-03-13
MXPA05007822A (es) 2006-02-10
CY1116215T1 (el) 2017-02-08
ES2541785T3 (es) 2015-07-24
EP1589993A4 (en) 2007-09-26
EP2857036B1 (en) 2016-12-21
IL169562A (en) 2016-04-21
EP2857036A1 (en) 2015-04-08
IL241315A0 (en) 2015-11-30
CN102580067A (zh) 2012-07-18
EP2444102B1 (en) 2015-04-08
JP2012102128A (ja) 2012-05-31
HK1169033A1 (en) 2013-01-18
EP2444102A1 (en) 2012-04-25
SI1589993T1 (sl) 2015-05-29
JP2006516645A (ja) 2006-07-06
ES2541952T3 (es) 2015-07-28
CY1116464T1 (el) 2017-03-15
IL241315A (en) 2016-12-29
US20040180419A1 (en) 2004-09-16
WO2004069190A2 (en) 2004-08-19
IL249389A0 (en) 2017-02-28
CA2814767A1 (en) 2004-08-19
EP1589993A2 (en) 2005-11-02
IL214440A (en) 2017-03-30

Similar Documents

Publication Publication Date Title
CY1116747T1 (el) Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης
CY1110013T1 (el) Συνδυασμοι για θεραπεια πολλαπλου μυελωματος
NO20050807L (no) Farmasoytiske sammensetninger som innbefatter dekstrometorfan og kinidin for behandling av neurologiske forstyrrelser
CY1110495T1 (el) Χρηση ενος αγωνιστη υποδοχεα nmda για τη θεραπεια εμβοης που προκαλειται απο κοχλιακη διεγερτοτοξικοτητα
NO20062504L (no) Sammensetninger og doseringsformer for forbedret absorpsjon
BR0109779A (pt) Soluções moleculares hidrofìlicas dispersas de carvedilol
ECSP045140A (es) Formulaciones farmacéuticas derivadas del platino
CY1108932T1 (el) Ενωσεις 4-(ετεροκυκλυλ)-βενζολοσουλφοξιμινης για την θεραπευτικη αγωγη φλεγμονωδων ασθενειων
CY1106771T1 (el) Φαρμακευτικη συνθεση η οποια χρησιμοποιειται για τον ελεγχο της γλυκοζης του αιματος σε ασθενεις με διαβητη τυπου 2
EE200200565A (et) Vaskulaarse kahjustava toimega kombinatsioonravi
CY1113116T1 (el) Υποκατεστημενες 4-αμινοκυκλοεξανολες
EA200501105A1 (ru) Фармацевтическая композиция
CY1110369T1 (el) Νεος συνεργιστικος συνδυασμος ο οποιος περιεχει ροφλουμιλαστη και φορμοτερολη
EA200501821A1 (ru) Фармацевтическая комбинация, содержащая модафинил и другой лекарственный препарат
BRPI0418157A (pt) terapêutica de tumores alogênicos
CY1107231T1 (el) Υποκατεστημενα παραγωγα 4-αμινοκυκλοεξανολης
MX2009003532A (es) Tratamiento de hiperbilirrubinemia infantil utilizando bajas dosis de estansoporfina.
ES2340842T3 (es) Anticuerpos anti-apc no neutralizantes.
NO20051159L (no) Preparater og fremgangsmater for behandling av sykdommer som viser feilsammensetting av aggregering av protein
DE60222383D1 (de) Medikament zur verabreichung von amifostin und verwandten wirkstoffen
DE50113974D1 (de) Kombinationspräparate von 3-n-formylhydroxylaminopropylphosphonsäurederivaten oder 3-n-acetylhydroxylaminopropylphosphonsäurederivaten mit speziellen pharmazeutischen wirkstoffen
ATE395936T1 (de) Dichloracetat in kombination mit kardioprotektiven oder hämodynamischen medikamenten
SE0203817D0 (sv) New composition
EA200500865A1 (ru) Синергетическая комбинация, включающая рофлумиласт и r,r-формотерол
ATE482217T1 (de) Dithiolopyrrolon-derivate zur behandlung von proliferativen störungen