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)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (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 (OSRAM)
EP (2) EP1007043B9 (OSRAM)
JP (1) JP2001508064A (OSRAM)
AT (1) ATE268598T1 (OSRAM)
AU (1) AU5813898A (OSRAM)
CA (1) CA2278507C (OSRAM)
DE (1) DE69824421T2 (OSRAM)
DK (1) DK1007043T3 (OSRAM)
ES (1) ES2223118T3 (OSRAM)
PT (1) PT1007043E (OSRAM)
WO (1) WO1998030219A1 (OSRAM)

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
GB9909064D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
GB0100889D0 (en) * 2001-01-12 2001-02-21 Oxford Glycosciences Uk Ltd Compounds
WO2000066105A2 (en) * 1999-04-30 2000-11-09 The Trustees Of Boston College Treatment of epilepsy with imino sugars
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
US6642038B1 (en) 1999-09-14 2003-11-04 Genzyme Glycobiology Research Institute, Inc. GlcNAc phosphotransferase of the lysosomal targeting pathway
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1974752B1 (en) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 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
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
US6905856B2 (en) * 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
US20030124652A1 (en) * 2001-12-21 2003-07-03 Novazyme Pharmaceuticals, Inc. Methods of producing high mannose glycoproteins in complex carbohydrate deficient cells
AU2003248960B2 (en) * 2002-07-17 2009-06-25 Idorsia Pharmaceuticals Ltd Piperidinetriol derivatives as inhibitors of glycosylceramidsynthase
US7985760B2 (en) * 2002-07-17 2011-07-26 Actelion Pharmaceuticals Ltd. Piperidinetriol derivatives as inhibitors of glycosyceramid synthase
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
US20060018882A1 (en) * 2004-06-21 2006-01-26 Kaemmerer William F Medical devices and methods for delivering compositions to cells
PT2857036T (pt) 2003-01-31 2017-03-13 Mount Sinai School Of Medicine Of N Y Univ Terapia combinada para o tratamento de distúrbios de deficiência proteica
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
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
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
US7884115B2 (en) * 2004-09-28 2011-02-08 Allergan, Inc. Methods and compositions 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
AU2006247065B8 (en) 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
CA2669347A1 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
WO2008070769A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
ES2629853T3 (es) * 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP2475376B1 (en) 2009-06-17 2016-03-30 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
EP1007043B1 (en) 2004-06-09
EP1491196A3 (en) 2005-03-16
ATE268598T1 (de) 2004-06-15
EP1007043B9 (en) 2004-12-22
EP1491196A2 (en) 2004-12-29
CA2278507C (en) 2007-08-28
DE69824421D1 (de) 2004-07-15
CA2278507A1 (en) 1998-07-16
PT1007043E (pt) 2004-10-29
AU5813898A (en) 1998-08-03
JP2001508064A (ja) 2001-06-19
DE69824421T2 (de) 2005-03-03
EP1007043A1 (en) 2000-06-14
DK1007043T3 (da) 2004-10-11
US5798366A (en) 1998-08-25

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