ES2223118T3 - Metodo para el tratamiento de enfermedades de almacenamiento lisosomal en las que esta implicado el snc. - Google Patents

Metodo para el tratamiento de enfermedades de almacenamiento lisosomal en las que esta implicado el snc.

Info

Publication number
ES2223118T3
ES2223118T3 ES98901673T ES98901673T ES2223118T3 ES 2223118 T3 ES2223118 T3 ES 2223118T3 ES 98901673 T ES98901673 T ES 98901673T ES 98901673 T ES98901673 T ES 98901673T ES 2223118 T3 ES2223118 T3 ES 2223118T3
Authority
ES
Spain
Prior art keywords
disease
storage
mice
dnj
cns
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
ES98901673T
Other languages
English (en)
Spanish (es)
Inventor
Frances M. University of Oxford PLATT
Gabrielle R. Neises
Raymond A. University of Oxford DWEK
Terry D. University of Oxford BUTTERS
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Monsanto Co
Original Assignee
Monsanto Co
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 Monsanto Co filed Critical Monsanto Co
Application granted granted Critical
Publication of ES2223118T3 publication Critical patent/ES2223118T3/es
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7012—Compounds having a free or esterified carboxyl group attached, directly or through a carbon chain, to a carbon atom of the saccharide radical, e.g. glucuronic acid, neuraminic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
ES98901673T 1997-01-13 1998-01-13 Metodo para el tratamiento de enfermedades de almacenamiento lisosomal en las que esta implicado el snc. Expired - Lifetime ES2223118T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/782,321 US5798366A (en) 1993-05-13 1997-01-13 Method for treatment of CNS-involved lysosomal storage diseases
US782321 1997-01-13

Publications (1)

Publication Number Publication Date
ES2223118T3 true ES2223118T3 (es) 2005-02-16

Family

ID=25125672

Family Applications (1)

Application Number Title Priority Date Filing Date
ES98901673T Expired - Lifetime ES2223118T3 (es) 1997-01-13 1998-01-13 Metodo para el tratamiento de enfermedades de almacenamiento lisosomal en las que esta implicado el snc.

Country Status (11)

Country Link
US (1) US5798366A (enExample)
EP (2) EP1491196A3 (enExample)
JP (1) JP2001508064A (enExample)
AT (1) ATE268598T1 (enExample)
AU (1) AU5813898A (enExample)
CA (1) CA2278507C (enExample)
DE (1) DE69824421T2 (enExample)
DK (1) DK1007043T3 (enExample)
ES (1) ES2223118T3 (enExample)
PT (1) PT1007043E (enExample)
WO (1) WO1998030219A1 (enExample)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US6610703B1 (en) 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
GB0100889D0 (en) 2001-01-12 2001-02-21 Oxford Glycosciences Uk Ltd Compounds
GB9909064D0 (en) * 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
WO2000066105A2 (en) * 1999-04-30 2000-11-09 The Trustees Of Boston College Treatment of epilepsy with imino sugars
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
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US20030124652A1 (en) * 2001-12-21 2003-07-03 Novazyme Pharmaceuticals, Inc. Methods of producing high mannose glycoproteins in complex carbohydrate deficient cells
US6905856B2 (en) 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
EP1534676B1 (en) * 2002-07-17 2012-09-12 Actelion Pharmaceuticals Ltd. Piperidinetriol derivatives as inhibitors of glycosylceramide synthase
JP4585851B2 (ja) * 2002-07-17 2010-11-24 アクテリオン ファーマシューティカルズ リミテッド グルコシルセラミドシンターゼの阻害剤としてのピペリジントリオール誘導体
WO2004055157A2 (en) * 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
GB0313677D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compound
GB0313678D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compounds
ES2344302T3 (es) * 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
DE102004029310A1 (de) * 2004-06-17 2005-12-29 Clariant Gmbh Hochkonzentrierte, wässrige Formulierungen von Oligo-und Polyestern
EP1773401B1 (en) * 2004-06-21 2013-01-02 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
WO2006037069A1 (en) * 2004-09-28 2006-04-06 Allergan, Inc. Compositions comprising nb-dnj, ne-dnj or d-glucaro-delta-lactam and their uses for the treatment of pain and other neurological conditions
US8500720B2 (en) * 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac 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
JP2010509344A (ja) * 2006-11-13 2010-03-25 ザイストール セラピューティクス， インコーポレイテッド ポンペ病を治療するための方法
US20080140056A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Method for delivering large molecules to the brain
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CA2731346A1 (en) * 2010-02-09 2011-08-09 The Hospital For Sick Children Compounds for the treatment of lysosomal storage diseases
EP3318277A1 (en) * 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB1555654A (en) * 1977-06-25 1979-11-14 Exxon Research Engineering Co Agricultural burner apparatus
US4065562A (en) * 1975-12-29 1977-12-27 Nippon Shinyaku Co., Ltd. Method and composition for reducing blood glucose levels
NO154918C (no) * 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
GB2020278B (en) * 1978-05-03 1983-02-23 Nippon Shinyaku Co Ltd Moranoline dervitives
US4849430A (en) * 1988-03-09 1989-07-18 Monsanto Company Method of inhibiting virus
US5011829A (en) * 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5030638A (en) * 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US6291657B1 (en) * 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives

Also Published As

Publication number Publication date
WO1998030219A1 (en) 1998-07-16
AU5813898A (en) 1998-08-03
CA2278507A1 (en) 1998-07-16
CA2278507C (en) 2007-08-28
EP1007043B9 (en) 2004-12-22
JP2001508064A (ja) 2001-06-19
EP1007043B1 (en) 2004-06-09
DE69824421D1 (de) 2004-07-15
EP1007043A1 (en) 2000-06-14
DK1007043T3 (da) 2004-10-11
DE69824421T2 (de) 2005-03-03
ATE268598T1 (de) 2004-06-15
EP1491196A3 (en) 2005-03-16
US5798366A (en) 1998-08-25
PT1007043E (pt) 2004-10-29
EP1491196A2 (en) 2004-12-29

Similar Documents

Publication Publication Date Title
ES2223118T3 (es) Metodo para el tratamiento de enfermedades de almacenamiento lisosomal en las que esta implicado el snc.
Abed Rabbo et al. Sphingolipid lysosomal storage diseases: from bench to bedside
Schulze et al. Lysosomal lipid storage diseases
Schulze et al. Sphingolipids and lysosomal pathologies
Rosenbaum et al. Niemann‐Pick type C disease: molecular mechanisms and potential therapeutic approaches
Platt et al. Substrate reduction therapy
ES2333561T3 (es) Derivados de 1-desoxi-galactonojirimicina y su uso en el tratamiento de enfermadades relacionadas con el almacenamiento lisosomal potenciando la alfa-galactosidasa a lisosomal.
Jeyakumar et al. Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis
US8252789B2 (en) Compositions and methods for treating lysosomal disorders
EP0912179B1 (en) Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
Reynolds et al. Ceramide synthesis and metabolism as a target for cancer therapy
Butters et al. Therapeutic applications of imino sugars in lysosomal storage disorders
Sawkar et al. Therapeutic strategies to ameliorate lysosomal storage disorders–a focus on Gaucher disease
Beck New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy
Ries Enzyme replacement therapy and beyond—in memoriam Roscoe O. Brady, MD (1923–2016)
Karten et al. Mechanisms and consequences of impaired lipid trafficking in Niemann–Pick type C1-deficient mammalian cells
KR102076585B1 (ko) 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물
Muro Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders
Eckhardt Pathology and current treatment of neurodegenerative sphingolipidoses
Wraith Advances in the treatment of lysosomal storage disease
Kaidonis et al. N-butyldeoxynojirimycin treatment restores the innate fear response and improves learning in mucopolysaccharidosis IIIA mice
Giacobini Drugs that target cholinesterases
Matassini et al. Carbohydrate‐Based Therapeutics for Lysosomal Storage Disorders
Walkley et al. Lysosomal diseases
Ajmal et al. Advanced drug delivery systems in the management of Gaucher disease