CY1117312T1 - Θεραπευτικη αγωγη συνουκλεϊνοπαθειων - Google Patents

Θεραπευτικη αγωγη συνουκλεϊνοπαθειων

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Publication number
CY1117312T1
CY1117312T1 CY20161100131T CY161100131T CY1117312T1 CY 1117312 T1 CY1117312 T1 CY 1117312T1 CY 20161100131 T CY20161100131 T CY 20161100131T CY 161100131 T CY161100131 T CY 161100131T CY 1117312 T1 CY1117312 T1 CY 1117312T1
Authority
CY
Cyprus
Prior art keywords
cochlecopenia
therapeutic treatment
treatment
synucleinopathies
subjects
Prior art date
Application number
CY20161100131T
Other languages
English (en)
Inventor
Michael Schlossmacher
Valerie Cullen
Lamya Shihabuddin
Seng H Cheng
Original Assignee
The Brigham And Women΄S Hospital, Inc.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40122277&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1117312(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by The Brigham And Women΄S Hospital, Inc. filed Critical The Brigham And Women΄S Hospital, Inc.
Publication of CY1117312T1 publication Critical patent/CY1117312T1/el

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    • 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)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4873Cysteine endopeptidases (3.4.22), e.g. stem bromelain, papain, ficin, cathepsin H
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/045Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/047Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates having two or more hydroxy groups, e.g. sorbitol
    • 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/12Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
    • A61K38/13Cyclosporins
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/488Aspartic endopeptidases (3.4.23), e.g. pepsin, chymosin, renin, cathepsin E
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
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    • AHUMAN NECESSITIES
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/02Local antiseptics
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22Cysteine endopeptidases (3.4.22)
    • C12Y304/22041Cathepsin F (3.4.22.41)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/23Aspartic endopeptidases (3.4.23)
    • C12Y304/23005Cathepsin D (3.4.23.5)

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  • Oncology (AREA)
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  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

Αυτή η εφεύρεση αφορά γενικώς τη θεραπευτική αγωγή συνουκλεϊνοπαθειών σε υποκείμενα τα οποία δεν είναι κλινικώς διαγνωσμένα με ασθένεια λυσοσωμικής αποθήκευσης, καθώς και σχετικές μεθόδους παρασκευής φαρμάκων και μεθόδους διαλογής.
CY20161100131T 2007-05-16 2016-02-16 Θεραπευτικη αγωγη συνουκλεϊνοπαθειων CY1117312T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US93046207P 2007-05-16 2007-05-16
US92955407P 2007-07-03 2007-07-03
EP08755799.7A EP2154969B1 (en) 2007-05-16 2008-05-16 Treatment of synucleinopathies

Publications (1)

Publication Number Publication Date
CY1117312T1 true CY1117312T1 (el) 2017-04-26

Family

ID=40122277

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20161100131T CY1117312T1 (el) 2007-05-16 2016-02-16 Θεραπευτικη αγωγη συνουκλεϊνοπαθειων

Country Status (18)

Country Link
US (4) US8454954B2 (el)
EP (2) EP3000320B1 (el)
JP (4) JP5291093B2 (el)
CN (2) CN101754682B (el)
AU (1) AU2008254774B2 (el)
BR (1) BRPI0811265A2 (el)
CA (1) CA2684946C (el)
CY (1) CY1117312T1 (el)
DK (1) DK2154969T3 (el)
ES (1) ES2559859T3 (el)
HK (2) HK1141406A1 (el)
HR (1) HRP20151409T1 (el)
HU (1) HUE027278T2 (el)
MX (1) MX353727B (el)
PL (1) PL2154969T3 (el)
PT (1) PT2154969E (el)
SI (1) SI2154969T1 (el)
WO (1) WO2008144591A2 (el)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101754682B (zh) 2007-05-16 2014-11-12 布里格姆妇女医院 突触核蛋白病的治疗
US20110064721A1 (en) * 2007-07-12 2011-03-17 The Uab Research Foundation Cathepsin-d neuroprotection
GB201011420D0 (en) * 2010-07-06 2010-08-18 United Arab Emirates Universit Method for diagnosis
US9290759B2 (en) * 2010-08-25 2016-03-22 The Trustees Of Columbia University In The City Of New York Optimized miRNA constructs
ES2912284T3 (es) 2010-12-08 2022-05-25 Us Health Pirazolopirimidinas sustituidas como activadoras de la glucocerebrosidasa
ES2745470T3 (es) * 2011-03-31 2020-03-02 Univ Iowa Res Found Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
EP2533051A1 (en) 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
AU2013263346B2 (en) * 2012-05-18 2018-08-23 The General Hospital Corporation Methods and compositions for treating amyloid deposits
CN104902923A (zh) * 2012-11-05 2015-09-09 建新公司 用于治疗蛋白质病的组合物和方法
EP2919759A4 (en) * 2012-11-14 2016-07-20 Ohio State Innovation Foundation MATERIALS AND METHODS FOR THE TREATMENT OF GLIOBLASTOMES
EP3783020A1 (en) * 2013-11-21 2021-02-24 F. Hoffmann-La Roche AG Anti-alpha-synuclein antibodies and methods of use
WO2016025533A2 (en) * 2014-08-11 2016-02-18 Whitehead Institute For Biomedical Research Compositions and methods for treating synucleinopathies
GB2558326B (en) * 2014-09-05 2021-01-20 Population Bio Inc Methods and compositions for inhibiting and treating neurological conditions
WO2016164474A1 (en) * 2015-04-06 2016-10-13 The Trustees Of Columbia University In The City Of New York Predictive value of combined genetic enzymatic and lipidomic data in disease risk for lewy body disease
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
GB201512203D0 (en) * 2015-07-13 2015-08-19 Lundbeck & Co As H Agents,uses and methods
BR112018008407A2 (pt) * 2015-10-28 2018-11-27 Univ Pennsylvania administração intratecal de vetores virais adenoassociados para terapia genética
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
AU2018346105C1 (en) 2017-10-03 2023-10-12 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3085964A1 (en) * 2018-01-12 2019-07-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
EP3787748A1 (en) * 2018-05-01 2021-03-10 OrfoNeuro ApS Treatment of neuronal ceroid lipofuscinosis
MX2021009305A (es) 2019-02-04 2021-11-04 Freeline Therapeutics Ltd Polinucleotidos.
EP3953378A1 (en) 2019-04-10 2022-02-16 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112022001123A2 (pt) 2019-07-24 2022-03-15 Stealth Biotherapeutics Inc O composto peptidomimétido (r)-2-amino-n-((s-l-(((s)-5-aminol-(3-benzil-1,2,4,-oxadiazol5-il)pentil)amino-3-(4-hidroxi-2,6-dimetilfenil)-1-oxopropan-2-il)-5-guanidinopentanamida no tratamento de doenças neurodegenerativas
EP4117628A4 (en) * 2020-03-10 2024-04-10 University of Cincinnati MATERIALS AND METHODS FOR THE TREATMENT OF GAUCHER’S DISEASE
AU2021316875A1 (en) 2020-07-29 2023-02-23 Freeline Therapeutics Limited Mutated beta-glucocerebrosidase with improved stability

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US20020168750A1 (en) 1988-12-23 2002-11-14 James Rasmussen Enzymatically active recombinant glucocerebrosidase
DE69031951T2 (de) 1989-11-16 1998-08-13 Cornell Res Foundation Inc Transformation von tierischen Hautzellen mit hilfe von Partikeln
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
TW360548B (en) 1993-04-08 1999-06-11 Powderject Res Ltd Products for therapeutic use
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
US5571787A (en) * 1993-07-30 1996-11-05 Myelos Corporation Prosaposin as a neurotrophic factor
BR9407956A (pt) 1993-10-25 1996-11-26 Canji Inc Composiçao farmacêutica vetor de express o de adenovirus recombinante e kit par reduzir a proliferaçao de células tumorais
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
DE69535703T2 (de) 1994-04-13 2009-02-19 The Rockefeller University AAV-vermittelte Zufuhr von DNA an Zellen des Nervensystems
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
JP2002531063A (ja) * 1998-10-23 2002-09-24 ローゲル ニッシェ アルツハイマー症の診断又は予後の方法
AU7856600A (en) 1999-10-04 2001-05-10 University Of Medicine And Dentistry Of New Jersey Novel carbamates and ureas
ATE310096T1 (de) 2000-09-18 2005-12-15 Genzyme Corp Expressionsvektoren mit hybriden ubiquitin- promotoren
PE20040079A1 (es) * 2002-04-03 2004-04-19 Novartis Ag Derivados de indolilmaleimida
JP5624256B2 (ja) * 2003-06-20 2014-11-12 ラプター・ファーマシューティカル・インコーポレイテッド 脳および他の組織への治療化合物の送達
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
CA2611011C (en) * 2005-06-08 2014-01-28 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
GB2429013C (en) * 2005-08-11 2012-11-28 Arpi Matossian-Rogers Peptides for treatment and diagnosis of autoimmunedisease
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
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
CN101754682B (zh) * 2007-05-16 2014-11-12 布里格姆妇女医院 突触核蛋白病的治疗
US20110064721A1 (en) * 2007-07-12 2011-03-17 The Uab Research Foundation Cathepsin-d neuroprotection

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