HUE052158T2 - Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez - Google Patents

Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez

Info

Publication number
HUE052158T2
HUE052158T2 HUE17720480A HUE17720480A HUE052158T2 HU E052158 T2 HUE052158 T2 HU E052158T2 HU E17720480 A HUE17720480 A HU E17720480A HU E17720480 A HUE17720480 A HU E17720480A HU E052158 T2 HUE052158 T2 HU E052158T2
Authority
HU
Hungary
Prior art keywords
arimoclomol
associated disorders
treating
glucocerebrosidase associated
treating glucocerebrosidase
Prior art date
Application number
HUE17720480A
Other languages
English (en)
Inventor
Anders Hinsby
Thomas Jensen
Cathrine Fog-Tonnesen
Nikolaj Petersen
Claus Bornaes
Original Assignee
Orphazyme As
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 Orphazyme As filed Critical Orphazyme As
Publication of HUE052158T2 publication Critical patent/HUE052158T2/hu

Links

Classifications

    • 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • A61P25/28Drugs 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
    • A61P3/00Drugs for disorders of the metabolism
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
HUE17720480A 2016-04-29 2017-04-28 Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez HUE052158T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
DKPA201670281 2016-04-29

Publications (1)

Publication Number Publication Date
HUE052158T2 true HUE052158T2 (hu) 2021-04-28

Family

ID=58645076

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE17720480A HUE052158T2 (hu) 2016-04-29 2017-04-28 Arimoklomol a glükocerebroszidázzal társult rendellenességek kezeléséhez

Country Status (20)

Country Link
US (3) US20190111041A1 (hu)
EP (2) EP3448382B1 (hu)
JP (2) JP6818045B2 (hu)
KR (2) KR102254140B1 (hu)
CN (1) CN109069496A (hu)
AU (2) AU2017255959B2 (hu)
BR (1) BR112018070653A2 (hu)
CA (1) CA3021900A1 (hu)
CY (1) CY1123717T1 (hu)
DK (1) DK3448382T3 (hu)
ES (1) ES2831764T3 (hu)
HR (1) HRP20210047T1 (hu)
HU (1) HUE052158T2 (hu)
IL (2) IL262411B (hu)
LT (1) LT3448382T (hu)
PT (1) PT3448382T (hu)
RS (1) RS61291B1 (hu)
RU (2) RU2750154C2 (hu)
SI (1) SI3448382T1 (hu)
WO (1) WO2017186919A1 (hu)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230226036A1 (en) * 2020-06-24 2023-07-20 Orphazyme A/S Arimoclomol for treating gaucher disease
JP2024500632A (ja) 2020-11-19 2024-01-10 ゼブラ デンマーク エー/エス アリモクロモルクエン酸塩及びその中間体の調製プロセス

Family Cites Families (126)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1989008661A1 (en) 1988-03-18 1989-09-21 The General Hospital Corporation Human heat shock factor
US5348945A (en) 1990-04-06 1994-09-20 Wake Forest University Method of treatment with hsp70
DK0700445T3 (da) 1993-06-04 2002-05-13 Whitehead Biomedical Inst Stressproteiner og anvendelser deraf
DK0751957T3 (da) 1994-03-21 2004-06-01 Univ Utrecht Peptid-fragmenter af mikrobielle stressproteiner og farmaceutisk sammensætning fremstillet deraf til behandling og forebyggelse af inflammatoriske sygdomme
US5985270A (en) 1995-09-13 1999-11-16 Fordham University Adoptive immunotherapy using macrophages sensitized with heat shock protein-epitope complexes
US5935576A (en) 1995-09-13 1999-08-10 Fordham University Compositions and methods for the treatment and prevention of neoplastic diseases using heat shock proteins complexed with exogenous antigens
US5837251A (en) 1995-09-13 1998-11-17 Fordham University Compositions and methods using complexes of heat shock proteins and antigenic molecules for the treatment and prevention of neoplastic diseases
HU222994B1 (hu) * 1995-11-02 2004-01-28 BIOREX Kutató és Fejlesztő Rt. Hidroxilaminszármazékok és azok alkalmazása sejtek molekuláris chaperon-termelésének fokozására alkalmas gyógyszerkészítmények előállítására
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
IN183280B (hu) 1996-09-20 1999-10-30 Univ New Mexico
US7157089B1 (en) 1996-11-26 2007-01-02 Stressgen Biotechnologies Corporation Immune responses using compositions containing stress proteins
US5830464A (en) 1997-02-07 1998-11-03 Fordham University Compositions and methods for the treatment and growth inhibition of cancer using heat shock/stress protein-peptide complexes in combination with adoptive immunotherapy
US6017540A (en) 1997-02-07 2000-01-25 Fordham University Prevention and treatment of primary and metastatic neoplastic diseases and infectious diseases with heat shock/stress protein-peptide complexes
US6007821A (en) 1997-10-16 1999-12-28 Fordham University Method and compositions for the treatment of autoimmune disease using heat shock proteins
US5948646A (en) 1997-12-11 1999-09-07 Fordham University Methods for preparation of vaccines against cancer comprising heat shock protein-peptide complexes
CZ20002669A3 (cs) 1998-01-23 2001-11-14 National Jewish Medical And Research Center Pouľití proteinů tepelného ąoku nebo molekuly nukleové kyseliny, která jej kóduje, přípravek tyto látky obsahující, způsob výběru léčby přecitlivělosti nebo nedostatečnosti průchodu dýchacích cest
EP2295065B1 (en) 1998-02-20 2013-10-09 The University of Miami Modified heat shock protein-antigenic peptide complex
JP4832640B2 (ja) 1998-03-27 2011-12-07 ガブリエル ミュルソフ Hsp70タンパク質の新規使用
FR2777890B1 (fr) 1998-04-22 2000-12-29 Roussy Inst Gustave Composes peptidiques d'hsp70 utiles dans l'immunotherapie du cancer
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US6475490B1 (en) 1998-10-19 2002-11-05 Fordham University Compositions and methods for promoting tissue repair using heat shock proteins
HU226617B1 (en) 1998-12-14 2009-04-28 Cytrx Corp Optically active pyridyl-4h-1,2,4-oxadiazine derivatives, and pharmaceutical composition containing the compound as active ingredient
HUP9900475D0 (en) 1999-02-26 1999-04-28 Biorex Kutato Fejlesztoe Kft O-(3-piperidino-2-hydroxy-1-propyl)-hiyroximic acid-halogenid derivative, it's use for treating insulin resistance, and pharmaceutical compositions containing them as active component
EP1218030A4 (en) 1999-09-10 2004-09-15 Univ Fordham METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF GRAFT REJECTS USING THERMAL SHOCK PROTEINS
US20020039583A1 (en) 1999-09-30 2002-04-04 Subjeck John R. Stress protein compositions and methods for prevention and treatment of cancer and infectious disease
US20030236300A1 (en) 1999-10-27 2003-12-25 Yale University Conductance of improperly folded proteins through the secretory pathway and related methods for treating disease
AU1582801A (en) 1999-11-05 2001-06-06 Board Of Regents Of The University Of Nebraska, The Methods and compositions for protection against bovine herpesvirus
US6964851B2 (en) 1999-12-07 2005-11-15 Stressgen Biotechnologies Corp. Compositions and methods for detecting stress-inducible proteins
GB9930443D0 (en) 1999-12-22 2000-02-16 King S College London Novel use of heat shock proteins
US20020127219A1 (en) 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
US20010034042A1 (en) 2000-01-20 2001-10-25 Srivastava Pramod K. Complexes of peptide-binding fragments of heat shock proteins and their use as immunotherapeutic agents
AU2001229597A1 (en) 2000-01-21 2001-07-31 University Of Connecticut Health Center Compositions and methods to treat neurodegenerative disorders
AU2001227960A1 (en) 2000-01-21 2001-07-31 University Of Connecticut Health Center Heat shock/stress protein complexes as vaccines against neurodegenerative disorders
HUP0001583A2 (hu) 2000-04-18 2002-11-28 BIOREX Kutató és Fejlesztő Rt. Egy piridin-1-oxid-származék és eljárás annak átalakítására gyógyászati hatású vegyületekké
WO2001097829A2 (en) 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020037290A1 (en) 2000-08-07 2002-03-28 Armen Garo H. Compositions comprising heat shock proteins or alpha(2) macroglobulin, antigenic molecules and saponins, and methods of use thereof
ES2326114T3 (es) 2000-09-13 2009-10-01 Multimmune Gmbh Un peptido de hsp70 estimulador de la actividad de las celulas asesinas naturales (nk) y los usos del mismo.
CA2422867A1 (en) 2000-09-15 2002-04-25 University Of Connecticut Health Center Improved formulations using heat shock/stress protein-peptide complexes
US20020172682A1 (en) 2000-10-20 2002-11-21 University Of Connecticut Health Center Using heat shock proteins to increase immune response
IT1319277B1 (it) 2000-10-24 2003-09-26 Chiesi Farma Spa Proteine di fusione utili per il trattamento di immunizzazione dellamalattia di alzheimer.
WO2002041921A2 (en) 2000-11-03 2002-05-30 The Board Of Regents Of The University Of Nebraska Compositions for protection against bovine viral diseases
EP1209226A3 (en) 2000-11-07 2002-06-05 GSF-Forschungszentrum für Umwelt und Gesundheit GmbH Maturation of dendritic cells by recombinant heat shock protein 70 (hsp70)
US20020142299A1 (en) 2001-01-09 2002-10-03 Davidson Beverly L. PTD-modified proteins
WO2002065989A2 (en) 2001-02-20 2002-08-29 Uab Research Foundation Aminoglycoside treatment for lysosomal storage diseases
EP1536829A4 (en) 2001-08-20 2006-05-31 Univ Connecticut Health Ct METHOD FOR PRODUCING COMPOSITIONS WITH HEAT SHOCK PROTEINS OR ALPHA-2-MACROGLOBULIN FOR THE TREATMENT OF CANCER AND INFECTION DISEASES
HU0103939D0 (en) 2001-09-27 2001-11-28 Biorex Kutato Fejlesztoe Kft Pharmaceutical composition containing methformin and n-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride
CA2461254A1 (en) 2001-09-27 2003-04-10 I.D.M. Immuno-Designed Molecules Polypeptides derived from inducible hsp70 and pharmaceutical compositions containing the same
GB0123756D0 (en) 2001-10-03 2001-11-21 King S College London A novel chaperone-type of adjuvant for vaccination - Basic 1
US7125843B2 (en) 2001-10-19 2006-10-24 Neose Technologies, Inc. Glycoconjugates including more than one peptide
ITMI20012573A1 (it) 2001-12-06 2003-06-06 D B P Dev Biotechonologica L P Gli acilsalicilati:una nuova classe di induttori di risposta heat shock
US7384936B2 (en) 2002-01-11 2008-06-10 Cytrx Corporation Carboxamidine derivatives and their use in the treatment of vascular diseases
WO2003061684A2 (en) 2002-01-24 2003-07-31 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Tumour treatment compositions comprising hsp70 and tumour necrosis factor
WO2003068941A2 (en) 2002-02-13 2003-08-21 Duke University Modulation of immune response by non-peptide binding stress response polypeptides
IL148401A0 (en) 2002-02-26 2002-09-12 Hadasit Med Res Service Hsp70-derived peptides and uses thereof in the diagnosis and treatment of autoimmune diseases
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
CA2483449A1 (en) 2002-04-25 2003-11-06 University Of Connecticut Health Center Using heat shock proteins to improve the therapeutic benefit of a non-vaccine treatment modality
US6984389B2 (en) 2002-04-25 2006-01-10 University Of Connecticut Health Center Using heat shock proteins to improve the therapeutic benefit of a non-vaccine treatment modality
RU2375077C2 (ru) 2002-05-02 2009-12-10 Юниверсити Оф Коннектикут Хелт Сентер Использование белков теплового шока для повышения эффективности терапий антителами
JP2006507272A (ja) 2002-05-02 2006-03-02 ユニバーシティー オブ コネティカット ヘルス センター 熱ショックタンパク質−ペプチド複合体またはα−2−マクログロブリン−ペプチド複合体を含むワクチンに対する免疫応答を増強するための熱ショックタンパク質およびα−2−マクログロブリンの使用
GB0216414D0 (en) 2002-07-15 2002-08-21 Novartis Ag Organic compounds
JP2006515165A (ja) 2002-09-16 2006-05-25 ジェネンテック・インコーポレーテッド 免疫関連疾患の治療のための新規組成物と方法
ES2396231T3 (es) 2003-05-21 2013-02-20 Biotech Tools S.A. Complejo peptídico
US7244616B2 (en) 2003-06-27 2007-07-17 Bayer Pharmaceuticals Corporation Use of molecular chaperones for the enhanced production of secreted, recombinant proteins in mammalian cells
HUP0303584A3 (en) * 2003-10-30 2009-12-28 Cytrx Corp Use of a hydroximic acid halide derivative in the treatment of neurodegenerative diseases
EP2145896A3 (en) 2003-11-12 2010-03-31 Alfa Biogene International B.V. Recovery of a heat shock protein
EP1691819A4 (en) 2003-11-25 2009-05-06 Sinai School Medicine THERAPY ON CHAPERON BASIS FOR NIEMANN PICK DISEASE
ES2324538T3 (es) 2003-12-05 2009-08-10 Multimmune Gmbh Anticuerpos anti-hsp terapeuticos y de diagnostico.
US20060009520A1 (en) 2004-05-12 2006-01-12 Tall Alan R Retinoid-based methods for altering macrophage cholesterol
WO2005120558A2 (en) 2004-05-25 2005-12-22 University Of Connecticut Health Center Methods for making compositions comprising heat shock proteins or alpha-2-macroglobulin for the treatment of cancer and infectious disease
AU2005312415B2 (en) 2004-12-10 2012-02-02 Angteq B.V. Heat shock proteins (HSP) and supraventricular arrhythmia
KR20080033242A (ko) 2005-06-08 2008-04-16 아미쿠스 세라퓨틱스, 인코포레이티드 리소좀 효소를 코딩하는 유전자의 돌연변이와 관련있는cns 질환의 치료 방법
WO2007041285A2 (en) 2005-09-29 2007-04-12 Viral Genetics, Inc. Complexes of inactivated pepsin fraction and heat shock protein
SI2395002T1 (sl) 2005-11-08 2014-10-30 Vertex Pharmaceuticals Incorporated Farmacevtski sestavek, vsebujoč heterociklični modulator prenašalcev z ATP-vezavno kaseto
US20080039400A1 (en) 2006-01-24 2008-02-14 Universiteit Utrecht Uu Holding B.V. Treatment and prevention of inflammatory bowel diseases
AU2007210159A1 (en) 2006-01-26 2007-08-09 Foldrx Pharmaceuticals, Inc. Compounds and methods for modulating protein trafficking
WO2007137239A2 (en) 2006-05-19 2007-11-29 The Scripps Research Institute Treatment of protein misfolding
KR101402554B1 (ko) 2006-06-23 2014-06-19 아미쿠스 세라퓨틱스, 인코포레이티드 β-글루코세레브로시다제의 활성 증진에 의한 신경계 장애 치료 방법
JP5599610B2 (ja) 2006-06-30 2014-10-01 スローン − ケッタリング インスティチュート フォー キャンサー リサーチ Hsp90の阻害による神経変性疾患の処置
WO2008021210A2 (en) 2006-08-11 2008-02-21 Combinatorx, Incorporated Methods and compositions for the treatment of neurodegenerative disorders
EP2057195A4 (en) 2006-08-29 2010-05-12 Forhumantech Co Ltd PHARMACEUTICAL COMPOSITION FOR THE SUPPRESSION OF APOPTOSIS AND METHOD OF ADMINISTERING THE SAME
TW200831479A (en) * 2006-09-26 2008-08-01 Cytrx Corp Pharmaceutical compositions and methods for treating diseases
WO2008070010A2 (en) 2006-12-01 2008-06-12 Cytrx Corporation Hydroxylamine derivatives for the treatment of stroke
EP2132309A4 (en) 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASES RELATED TO GLYCOGEN STORAGE
EP2155197A4 (en) 2007-03-09 2011-10-12 Link Medicine Corp TREATMENT OF LYSOSOMAL STORAGE DISEASES
GB0705626D0 (en) 2007-03-23 2007-05-02 Royal Veterinary College Method for enhancing sperm survival
WO2008127680A2 (en) 2007-04-11 2008-10-23 The Jackson Laboratory Diagnosis and treatment of diseases caused by misfolded proteins
MX2009011055A (es) 2007-04-13 2010-01-13 Amicus Therapeutics Inc Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos.
DK2481400T3 (da) 2007-04-25 2014-09-29 Opko Ip Holdings Ii Inc Orale præparater med kontrolleret frigivelse omfattende en vitamin D-forbindelse og en voksagtig bærer
US9056101B2 (en) 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
TW200901958A (en) 2007-05-04 2009-01-16 Cytrx Corp Diabetic wound healing
GB0712494D0 (en) 2007-06-27 2007-08-08 Isis Innovation Substrate reduction therapy
US7678764B2 (en) 2007-06-29 2010-03-16 Johnson & Johnson Regenerative Therapeutics, Llc Protein formulations for use at elevated temperatures
US8742068B2 (en) 2007-07-06 2014-06-03 Universiteit Utrecht Holding B.V. Treatment and prevention of inflammatory diseases and autoimmune diseases
WO2009036349A1 (en) 2007-09-12 2009-03-19 Anaphore, Inc. Hsp70-based treatment for autoimmune diseases
EP2252288A1 (en) 2007-11-21 2010-11-24 Summit Corporation Plc Treatment of protein folding disorders
GB2466912B (en) 2007-11-29 2012-01-04 Hospital For Sick Children Compositions and methods for treating lysosomal disorders
ATE555191T1 (de) 2008-01-30 2012-05-15 Geron Corp Synthetische oberflächen zur kultivierung von zellen in chemisch definierten medien
WO2009095452A1 (en) 2008-01-31 2009-08-06 Crystax Pharmaceuticals, S.L. Crystal structure of the atpase domain of proteins of the hsp70 family
CA2712593A1 (en) 2008-02-01 2009-08-13 The Scripps Research Institute Methods for treating a condition characterized by dysfunction in protein homeostasis
EP3778652A1 (en) 2008-05-07 2021-02-17 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
WO2009137796A2 (en) 2008-05-08 2009-11-12 Northwestern University Method of regulating the heat shock response
GB0809360D0 (en) 2008-05-22 2008-07-02 Isis Innovation Calcium modulation
ES2963910T3 (es) * 2008-06-26 2024-04-03 Zevra Denmark As Uso de Hsp70 como regulador de la actividad enzimática
EP2323652A2 (en) 2008-08-06 2011-05-25 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
WO2010022461A1 (en) 2008-08-29 2010-03-04 Children, Youth And Women's Health Service Sulphated sugars to increase activity of sulphatases en lysosomal storage disorders
CA2736545A1 (en) 2008-09-29 2010-04-01 Vertex Pharmaceuticals Incorporated Dosage units of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
WO2010053655A2 (en) 2008-11-07 2010-05-14 University Of Kansas Therapeutic methods with withaferin a and analogs
EP2391229B1 (en) 2009-01-29 2015-07-15 Alfa Biogene International B.V. Functional food product comprising heat shock protein or a hydrolysate thereof from alfalfa
EP2218458A1 (en) 2009-02-13 2010-08-18 Fondazione Telethon Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof
WO2010102988A1 (en) 2009-03-10 2010-09-16 Alfa Biogene International B.V. Skin care product
GB0906159D0 (en) 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
US8785168B2 (en) 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
MX2012001268A (es) 2009-07-28 2012-09-12 Shire Human Genetic Therapies Composiciones y metodos para tratar enfermedad de gaucher.
US20120156180A1 (en) 2009-08-10 2012-06-21 The Board Of Trustees Of The University Of Illinois Compositions and methods for the treatment of krabbe and other neurodegenerative diseases
US20110081428A1 (en) 2009-09-16 2011-04-07 The Buck Institute For Age Research Use of thioflavin-like compounds to increase life span and/or health span
US8609416B2 (en) 2009-12-18 2013-12-17 Ventria Bioscience Methods and compositions comprising heat shock proteins
US20120009125A1 (en) * 2010-07-06 2012-01-12 Lombard Jay L Apoe4 and apoj biomarker-based prevention and treatment of dementia
WO2012012656A2 (en) 2010-07-21 2012-01-26 University Of South Florida Materials and methods for treating neurodegenerative diseases
EP2646044B1 (en) 2010-11-30 2019-08-28 Orphazyme A/S Methods for increasing intracellular activity of hsp70
WO2013006076A1 (en) 2011-07-04 2013-01-10 New York University The use of intranasally administered hsp70 protein to treat neurodegenerative diseases
EP2831039A1 (en) 2012-03-28 2015-02-04 The U.S.A. as represented by the Secretary, Department of Health and Human Services Salicylic acid derivatives useful as glucocerebrosidase activators
AU2013337354A1 (en) * 2012-11-05 2015-05-21 Genzyme Corporation Compositions and methods for treating proteinopathies
JP6571527B2 (ja) 2012-11-21 2019-09-04 ウーハン ワイゼットワイ バイオファルマ カンパニー リミテッドWuhan Yzy Biopharma Co., Ltd. 二重特異性抗体
WO2015119989A1 (en) * 2014-02-04 2015-08-13 New York University Progranulin (pgrn) and its derivatives for diagnosis and treatment of lysosomal storage diseases
AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
BR112017007025A2 (pt) * 2014-10-10 2017-12-12 Univ Virginia Commonwealth sulfatos de colesterol oxigenados para terapia de distúrbios causados por pelo menos um de atividade de leptina atenuada e um distúrbio de armazenamento de lipídio

Also Published As

Publication number Publication date
WO2017186919A1 (en) 2017-11-02
JP2019518725A (ja) 2019-07-04
RS61291B1 (sr) 2021-02-26
KR20190003523A (ko) 2019-01-09
EP3448382A1 (en) 2019-03-06
RU2018138791A3 (hu) 2020-06-25
RU2018138791A (ru) 2020-05-29
AU2017255959B2 (en) 2020-10-15
HRP20210047T1 (hr) 2021-04-16
CN109069496A (zh) 2018-12-21
KR102254140B1 (ko) 2021-05-24
AU2017255959A1 (en) 2018-11-15
CY1123717T1 (el) 2022-05-27
RU2021117465A (ru) 2021-07-22
ES2831764T3 (es) 2021-06-09
EP3448382B1 (en) 2020-10-14
BR112018070653A2 (pt) 2019-02-05
RU2750154C2 (ru) 2021-06-22
LT3448382T (lt) 2021-04-12
JP2021059586A (ja) 2021-04-15
US20200289497A1 (en) 2020-09-17
SI3448382T1 (sl) 2021-03-31
US20220133707A1 (en) 2022-05-05
US11253505B2 (en) 2022-02-22
AU2020277270A1 (en) 2020-12-24
IL262411A (en) 2018-12-31
IL280227A (en) 2021-03-01
PT3448382T (pt) 2020-11-20
DK3448382T3 (da) 2020-12-07
CA3021900A1 (en) 2017-11-02
EP3782624A1 (en) 2021-02-24
IL262411B (en) 2021-02-28
US20190111041A1 (en) 2019-04-18
JP6818045B2 (ja) 2021-01-20
KR20210059796A (ko) 2021-05-25
AU2020277270B2 (en) 2023-02-23

Similar Documents

Publication Publication Date Title
IL251721A0 (en) Preparations and methods for the treatment of diseases of the central nervous system
IL279308A (en) Methods for the treatment of heptidine-mediated disorders
HK1256638A1 (zh) 用於治療骨髓增生性病症的方法
LT3206493T (lt) Kompozicijos ir būdai, skirti cns sutrikimams gydyti
PT3224269T (pt) Composições e métodos para tratamento de distúrbios do snc
HK1256036A1 (zh) 治療骨髓增生性障礙的方法
IL266530A (en) Fibrosis treatment
IL269082A (en) Methods for treating lysosomal disorders
PT3119911T (pt) Compostos para uso no tratamento da doença de alzheimer em pacientes apoe4+/+
EP3618783C0 (en) DEVICES FOR TREATING EYELIDS
HK1256298A1 (zh) 用於治療和預防神經退化性病症的組合物和方法
EP3262065C0 (en) METHODS AND COMPOSITIONS FOR THE TREATMENT OF DYSTROGLYCANOPATHY DISEASES
IL280227A (en) Arimoclomol for the treatment of glucocerebrosidase-related disorders
IL271211A (en) Migraine treatment
PT3420084T (pt) Nova abordagem para tratamento de disfunções inflamatórias
GB201413532D0 (en) Combination therapy for treating hearing disorders
GB201602011D0 (en) Treating skin disorders
GB201517995D0 (en) Products for treating psychogenic pain disorders