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CY1116464T1 - Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης - Google Patents

Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Info

Publication number
CY1116464T1
CY1116464T1 CY20151100594T CY151100594T CY1116464T1 CY 1116464 T1 CY1116464 T1 CY 1116464T1 CY 20151100594 T CY20151100594 T CY 20151100594T CY 151100594 T CY151100594 T CY 151100594T CY 1116464 T1 CY1116464 T1 CY 1116464T1
Authority
CY
Cyprus
Prior art keywords
combination therapy
protein
therapeutic treatment
protein deficiency
deficiency disorders
Prior art date
Application number
CY20151100594T
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 CY1116464T1 publication Critical patent/CY1116464T1/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, 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)

Abstract

Η παρούσα αίτηση παρέχει μεθόδους βελτίωσης θεραπείας υποκατάστασης πρωτεΐνης μέσω συνδυασμού θεραπείας υποκατάστασης πρωτεΐνης με ενεργές τοποειδικές χαπερόνες (ASSC), ώστε να αυξηθεί η σταθερότητα και η αποτελεσματικότητα της εκάστοτε χορηγούμενης πρωτεΐνης. Η αίτηση περαιτέρω παρέχει συνθέσεις που περιλαμβάνουν την κεκαθαρμένη πρωτεΐνη και μία ASSC, και μεθόδους θεραπευτικής αγωγής μέσω χορήγησης των συνθέσεων.
CY20151100594T 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης CY1116464T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44413603P 2003-01-31 2003-01-31
EP20110006495 EP2444102B1 (en) 2003-01-31 2004-02-02 Combination therapy for treating protein deficiency disorders

Publications (1)

Publication Number Publication Date
CY1116464T1 true CY1116464T1 (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
CY20151100593T CY1116463T1 (el) 2003-01-31 2015-07-08 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
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 (2)

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
CY20151100593T CY1116463T1 (el) 2003-01-31 2015-07-08 Θεραπεια συνδυασμου για τη θεραπευτικη αγωγη διαταραχων ανεπαρκειας πρωτεϊνης

Family Applications After (2)

Application Number Title Priority Date Filing Date
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

Country Status (16)

Country Link
US (4) US20040180419A1 (el)
EP (6) EP1589993B1 (el)
JP (5) JP5589165B2 (el)
CN (4) CN102580067A (el)
BR (1) BRPI0407001A (el)
CA (3) CA2814767C (el)
CY (5) CY1116215T1 (el)
DK (5) DK1589993T3 (el)
ES (6) ES2545619T3 (el)
HK (4) HK1158955A1 (el)
HU (2) HUE033381T2 (el)
IL (6) IL169562A (el)
MX (2) MX344587B (el)
PT (5) PT1589993E (el)
SI (5) SI2444102T1 (el)
WO (1) WO2004069190A2 (el)

Families Citing this family (56)

* 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
US20020095135A1 (en) * 2000-06-19 2002-07-18 David Meeker 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
SI2444102T1 (sl) * 2003-01-31 2015-08-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
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
AU2006247065B8 (en) 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin 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
EP2361613B1 (en) * 2006-02-07 2020-08-12 Shire Human Genetic Therapies, Inc. 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
EP2040548B1 (en) 2006-06-23 2012-05-02 Amicus Therapeutics, Inc. Method for the treatment of neurological disorders by enhancing the activity of beta -glucocerebrosidase
CA2657164A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic light toxicity
AU2007277032A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Opsin stabilizing compounds and methods of use
CA2657238A1 (en) * 2006-07-27 2008-01-31 University Of Florida Research Foundation, Inc. Compositions and methods for treating or preventing ophthalmic disease
EP2150254A4 (en) * 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
US9999618B2 (en) 2007-04-26 2018-06-19 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
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
CN103432566A (zh) 2008-12-16 2013-12-11 建新公司 寡糖-蛋白缀合物
US8450345B2 (en) 2009-02-23 2013-05-28 The Chancellor, Masters And Scholars Of The University Of Oxford Iminosugars and methods of treating viral diseases
WO2010099064A1 (en) * 2009-02-24 2010-09-02 United Therapeutics Corporation Iminosugars and methods of treating arenaviral infections
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
PL3354277T3 (pl) 2009-07-28 2021-12-13 Takeda Pharmaceutical Company Limited Kompozycje i sposoby leczenia choroby gauchera
CA2772875A1 (en) * 2009-09-04 2011-03-10 United Therapeutics Corporation Methods of treating orthomyxoviral infections
JP5575246B2 (ja) * 2009-09-04 2014-08-20 ユナイテッド セラピューティクス コーポレイション ポックスウイルス感染の治療方法
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
TW201117810A (en) * 2009-10-01 2011-06-01 Baylor Res Inst Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
DK3103469T3 (en) 2010-06-25 2021-02-22 Shire Human Genetic Therapies Indgivelse af terapeutiske midler til centralnervesystemet
NZ702808A (en) * 2010-06-25 2016-08-26 Shire Human Genetic Therapies Methods and compositions for cns delivery of iduronate-2-sulfatase
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
KR102076585B1 (ko) * 2011-12-22 2020-02-12 센토제너 아이피 게엠베하 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물
CN104519905A (zh) 2012-03-02 2015-04-15 夏尔人类遗传性治疗公司 用于治疗iii型戈谢病的组合物和方法
MX349992B (es) * 2012-03-07 2017-08-22 Amicus Therapeutics Inc Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe.
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
SG10202003753PA (en) 2014-09-30 2020-05-28 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2017117407A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
LT3397273T (lt) 2015-12-30 2021-06-25 Amicus Therapeutics, Inc. Patobulinta rūgštinė alfa-gliukozidazė pompe ligos gydymui
BR112018070189A2 (pt) 2016-03-30 2019-02-19 Amicus Therapeutics, Inc. método para seleção de proteínas recombinantes ricas em m6p
BR112018069965A2 (pt) 2016-03-30 2019-01-29 Amicus Therapeutics Inc formulações compreendendo alfa-glucosidase ácida recombinante
EP4389213A3 (en) 2016-07-19 2024-08-21 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
CA3056223A1 (en) 2017-03-27 2018-10-04 Regeneron Pharmaceuticals, Inc. Sterilization methods for medical products and sterilized medical products
CN119113091A (zh) 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
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
WO2019023648A1 (en) 2017-07-28 2019-01-31 Applied Therapeutics Inc. COMPOSITIONS AND METHODS FOR TREATING GALACTOSEMIA
KR20200079280A (ko) * 2017-10-26 2020-07-02 샤이어 휴먼 지네틱 테라피즈 인크. 글루코세레브로시다제 및 이소파고민을 포함하는 제제
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
US20230110129A1 (en) * 2019-04-25 2023-04-13 Takeda Pharmaceutical Company Limited 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.

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
DK0585368T3 (da) 1991-04-25 1998-03-16 Univ Brown Res Found Implanterbart biokompatibelt immunisolatorisk vehikel til afgivelse af udvalgte terapeutiske produkter
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
DE69332105T2 (de) 1992-09-29 2003-03-06 Inhale Therapeutic Systems, San Carlos Pulmonale abgabe von aktiven fragmenten des parathormons
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
ATE366741T1 (de) 1993-05-27 2007-08-15 Aventis Pharma Inc Topologisch getrennte, kodierende festphasen- bibliotheken
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
DE69835367T2 (de) * 1997-10-29 2007-08-02 Genzyme Corp., Framingham Gentherapie für gaucher-krankheit
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
CZ20011671A3 (cs) 1998-11-13 2001-10-17 Cyclacel Limited Peptidová nosná skupina pro transport
ES2677343T3 (es) 1998-12-07 2018-08-01 Genzyme Corporation Tratamiento de la enfermedad de Pompe
JP2003505430A (ja) 1999-07-26 2003-02-12 ジー・ディー・サール・アンド・カンパニー デオキシノジリマイシンの長鎖ｎ−アルキル誘導体およびグルコセレブロシダーゼ酵素の糖脂質異常蓄積疾患の治療用の薬剤の製造のための使用
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
WO2004037373A2 (en) 2002-10-21 2004-05-06 The Scripps Research Institute CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF β-GLUCOSIDASE
SI2444102T1 (sl) * 2003-01-31 2015-08-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
AU2005211775B2 (en) 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
AU2006247065B8 (en) * 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES

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