US20190111041A1 - Arimoclomol for treating glucocerebrosidase associated disorders - Google Patents
Arimoclomol for treating glucocerebrosidase associated disorders Download PDFInfo
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- US20190111041A1 US20190111041A1 US16/092,070 US201716092070A US2019111041A1 US 20190111041 A1 US20190111041 A1 US 20190111041A1 US 201716092070 A US201716092070 A US 201716092070A US 2019111041 A1 US2019111041 A1 US 2019111041A1
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- pharmaceutical ingredient
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- 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
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/4545—Non 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- A61P25/28—Drugs 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
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (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)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
DKPA201670281 | 2016-04-29 | ||
DKPA201670281 | 2016-04-29 | ||
PCT/EP2017/060205 WO2017186919A1 (en) | 2016-04-29 | 2017-04-28 | Arimoclomol for treating glucocerebrosidase associated disorders |
Related Parent Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/EP2017/060205 A-371-Of-International WO2017186919A1 (en) | 2016-04-29 | 2017-04-28 | Arimoclomol for treating glucocerebrosidase associated disorders |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US16/879,198 Continuation US11253505B2 (en) | 2016-04-29 | 2020-05-20 | Arimoclomol for treating glucocerebrosidase associated disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
US20190111041A1 true US20190111041A1 (en) | 2019-04-18 |
Family
ID=58645076
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US16/092,070 Abandoned US20190111041A1 (en) | 2016-04-29 | 2017-04-28 | Arimoclomol for treating glucocerebrosidase associated disorders |
US16/879,198 Active 2037-05-18 US11253505B2 (en) | 2016-04-29 | 2020-05-20 | Arimoclomol for treating glucocerebrosidase associated disorders |
US17/574,239 Pending US20220133707A1 (en) | 2016-04-29 | 2022-01-12 | Arimoclomol for treating glucocerebrosidase associated disorders |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US16/879,198 Active 2037-05-18 US11253505B2 (en) | 2016-04-29 | 2020-05-20 | Arimoclomol for treating glucocerebrosidase associated disorders |
US17/574,239 Pending US20220133707A1 (en) | 2016-04-29 | 2022-01-12 | Arimoclomol for treating glucocerebrosidase associated disorders |
Country Status (20)
Country | Link |
---|---|
US (3) | US20190111041A1 (ko) |
EP (2) | EP3782624A1 (ko) |
JP (2) | JP6818045B2 (ko) |
KR (2) | KR102254140B1 (ko) |
CN (1) | CN109069496A (ko) |
AU (2) | AU2017255959B2 (ko) |
BR (1) | BR112018070653A2 (ko) |
CA (1) | CA3021900A1 (ko) |
CY (1) | CY1123717T1 (ko) |
DK (1) | DK3448382T3 (ko) |
ES (1) | ES2831764T3 (ko) |
HR (1) | HRP20210047T1 (ko) |
HU (1) | HUE052158T2 (ko) |
IL (2) | IL262411B (ko) |
LT (1) | LT3448382T (ko) |
PT (1) | PT3448382T (ko) |
RS (1) | RS61291B1 (ko) |
RU (2) | RU2750154C2 (ko) |
SI (1) | SI3448382T1 (ko) |
WO (1) | WO2017186919A1 (ko) |
Cited By (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US11707456B2 (en) | 2020-11-19 | 2023-07-25 | Kempharm Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL299250A (en) * | 2020-06-24 | 2023-02-01 | Kempharm Denmark As | Arimoclomol for the treatment of Gaucher disease |
Family Cites Families (126)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JPH03503410A (ja) | 1988-03-18 | 1991-08-01 | ザ・ジェネラル・ホスピタル・コーポレーション | ヒト熱ショック因子 |
US5348945A (en) | 1990-04-06 | 1994-09-20 | Wake Forest University | Method of treatment with hsp70 |
PT700445E (pt) | 1993-06-04 | 2002-07-31 | Whitehead Biomedical Inst | Proteinas de stress e suas utilizacoes |
ATE260295T1 (de) | 1994-03-21 | 2004-03-15 | Univ Utrecht | Peptidfragmente von mikrobiellen stressproteinen und daraus hergestellte pharmazeutische zusammensetzungen für die behandlung und vorbeugung von entzündungserkrankungen |
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 |
CA2265935C (en) | 1996-09-20 | 2006-09-05 | The University Of New Mexico | Heat shock protein complexes |
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 |
IL137404A0 (en) | 1998-01-23 | 2001-07-24 | Nat Jewish Med & Res Center | Method for treating inflammatory diseases using heat shock proteins |
ATE359084T1 (de) | 1998-02-20 | 2007-05-15 | Univ Miami | Modifizierter hitzeschockprotein/peptidantigen komplex |
WO1999049881A2 (de) | 1998-03-27 | 1999-10-07 | Gabriele Multhoff | Verwendung von hsp70 protein |
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 |
WO2001017554A1 (en) | 1999-09-10 | 2001-03-15 | Fordham University | Methods and compositions for the treatment and prevention of graft rejection using heat 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 |
AU2001227960A1 (en) | 2000-01-21 | 2001-07-31 | University Of Connecticut Health Center | Heat shock/stress protein complexes as vaccines against neurodegenerative disorders |
WO2001052877A1 (en) | 2000-01-21 | 2001-07-26 | University Of Connecticut Health Center | Compositions and methods to treat 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 |
AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
AU2001277961A1 (en) | 2000-08-07 | 2002-02-18 | Antigenics, Llc | Compositions comprising heat shock proteins or alpha(2)macroglobulin, antigenic molecules and saponins, and methods of use thereof |
ATE430161T1 (de) | 2000-09-13 | 2009-05-15 | Multimmune Gmbh | Peptid aus hsp70 welches nk zellaktivtät stimuliert und seine verwendung |
WO2002032923A2 (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 |
CA2438750A1 (en) | 2001-02-20 | 2002-08-29 | Uab Research Foundation | Aminoglycoside treatment for lysosomal storage diseases |
IL160511A0 (en) | 2001-08-20 | 2004-07-25 | Univ Connecticut Health Ct | Methods for preparing compositions comprising heat shock proteins or alpha-2-macroglobulin useful for the treatment of cancer and infectious disease |
US20050267020A1 (en) | 2001-09-27 | 2005-12-01 | Olivier Faure | Polypeptides derived from inducible hsp70 and pharmaceutical compositions containing the same |
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 |
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 |
NZ533795A (en) | 2002-01-11 | 2006-03-31 | Biorex Kutato Fejlesztoe Kft | Carboxamidine derivatives and their use in the treatment of vascular diseases |
AU2003218639A1 (en) | 2002-01-24 | 2003-09-02 | Universiteit Gent | Tumour treatment compositions comprising hsp70 and tumour necrosis factor |
JP4632664B2 (ja) | 2002-02-13 | 2011-02-16 | デューク ユニバーシティ | 非ペプチド結合ストレス応答性ポリペプチドによる免疫応答の調節 |
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 |
US7658916B2 (en) | 2002-04-05 | 2010-02-09 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density |
IL164799A0 (en) | 2002-04-25 | 2005-12-18 | Univ Connecticut | 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 |
WO2004035602A2 (en) | 2002-05-02 | 2004-04-29 | University Of Connecticut Health Center | Using heat shock proteins and alpha-2-macroglobulins to increase immune response to vaccines |
US20060093612A1 (en) | 2002-05-02 | 2006-05-04 | Srivastava Pramod K | Use of heat shock proteins to enhance efficacy of antibody therapeutics |
GB0216414D0 (en) | 2002-07-15 | 2002-08-21 | Novartis Ag | Organic compounds |
CA2498274A1 (en) | 2002-09-16 | 2004-03-25 | Genentech, Inc. | Compositions and methods for the diagnosis of immune related diseases using pro7 |
US8436136B2 (en) | 2003-05-21 | 2013-05-07 | Biotech Tools S.A. | Peptide complex |
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 |
DE60335549D1 (de) | 2003-11-12 | 2011-02-10 | Alfa Biogene Internat B V | Gewinnung von Hitzeschockproteinen |
JP2007514661A (ja) | 2003-11-25 | 2007-06-07 | マウント シナイ スクール オブ メディスン オブ ニューヨーク ユニバーシティー | ニーマンピック病のシャペロンに基づく治療方法 |
ES2484340T3 (es) | 2003-12-05 | 2014-08-11 | Multimmune Gmbh | Anticuerpos anti hsp70 terapéuticos y diagnósticos |
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 |
JP2008523055A (ja) | 2004-12-10 | 2008-07-03 | ゼルニケ ビジネス サポート ビー.ヴィー. | 熱ショックタンパク質(hsp)および上室性不整脈 |
EP1888068B1 (en) | 2005-06-08 | 2014-05-21 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
WO2007041285A2 (en) | 2005-09-29 | 2007-04-12 | Viral Genetics, Inc. | Complexes of inactivated pepsin fraction and heat shock protein |
HUE032640T2 (en) | 2005-11-08 | 2017-10-30 | Vertex Pharma | Heterocyclic modulator of ATP-binding cassette transcripts |
US20080039400A1 (en) | 2006-01-24 | 2008-02-14 | Universiteit Utrecht Uu Holding B.V. | Treatment and prevention of inflammatory bowel diseases |
WO2007089548A2 (en) | 2006-01-26 | 2007-08-09 | Foldrx Pharmaceuticals, Inc. | Compounds and methods for modulating protein trafficking |
CA2653052A1 (en) | 2006-05-19 | 2007-11-29 | The Scripps Research Institute | Treatment of protein misfolding |
JP5303458B2 (ja) | 2006-06-23 | 2013-10-02 | アミークス セラピューティックス インコーポレイテッド | β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法 |
US10336757B2 (en) | 2006-06-30 | 2019-07-02 | Sloan-Kettering Institute For Cancer Research | Treatment of neurodegenerative diseases through inhibition of HSP90 |
WO2008021210A2 (en) | 2006-08-11 | 2008-02-21 | Combinatorx, Incorporated | Methods and compositions for the treatment of neurodegenerative disorders |
WO2008047243A2 (en) | 2006-08-29 | 2008-04-24 | Forhumantech. Co., Ltd. | Pharmaceutical composition for suppression of apoptosis and method for delivering the same |
WO2008039514A1 (en) * | 2006-09-26 | 2008-04-03 | Cytrx Corporation | Pharmaceutical compositions and methods for treating diseases associated with neurodegeneration |
EP2089033A2 (en) | 2006-12-01 | 2009-08-19 | CytRx Corporation | Stroke recovery |
CA2680063A1 (en) | 2007-02-23 | 2008-08-28 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating glycogen storage diseases |
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. |
EP3542792B1 (en) | 2007-04-25 | 2023-06-28 | EirGen Pharma Ltd. | Controlled release 25-hydroxyvitamin d |
EP2150254A4 (en) | 2007-04-26 | 2010-11-10 | Amicus Therapeutics Inc | DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES |
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 |
CN101801402B (zh) | 2007-07-06 | 2013-08-28 | 乌得勒支大学控股有限公司 | 炎性疾病和自身免疫疾病的治疗和预防 |
WO2009036349A1 (en) | 2007-09-12 | 2009-03-19 | Anaphore, Inc. | Hsp70-based treatment for autoimmune diseases |
WO2009066069A1 (en) | 2007-11-21 | 2009-05-28 | Summit Corporation Plc | Treatment of protein folding disorders |
WO2009073564A1 (en) | 2007-11-29 | 2009-06-11 | The Hospital For Sick Children | Compositions and methods for treating lysosomal disorders |
DK2247716T3 (da) | 2008-01-30 | 2012-05-29 | Geron Corp | Syntetiske overflader til dyrkning af celler i kemisk defineret medie |
WO2009095452A1 (en) | 2008-01-31 | 2009-08-06 | Crystax Pharmaceuticals, S.L. | Crystal structure of the atpase domain of proteins of the hsp70 family |
WO2009100037A1 (en) | 2008-02-01 | 2009-08-13 | The Scripps Research Institute | Methods for treating a condition characterized by dysfunction in protein homeostasis |
PL2279210T3 (pl) | 2008-05-07 | 2017-10-31 | Biomarin Pharm Inc | Lizosomalne peptydy kierujące i ich zastosowania |
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 |
CA2728363C (en) * | 2008-06-26 | 2019-02-19 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
US20110237538A1 (en) | 2008-08-06 | 2011-09-29 | 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 |
MX2011003249A (es) | 2008-09-29 | 2011-05-19 | Vertex Pharma | Unidades de dosificacion del acido 3-(6-(1-(2,2-difluorobenzo[d][1 ,3]dioxol-5-il)ciclopropancarboxamido)-3-metilpiridin-2-il)benzoi co. |
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 |
EP3075386B1 (en) | 2009-06-17 | 2019-10-16 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
ES2668079T3 (es) | 2009-07-28 | 2018-05-16 | Shire Human Genetic Therapies | Composiciones y métodos para tratar la 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 |
WO2012006329A2 (en) * | 2010-07-06 | 2012-01-12 | Genomind, Llc | Apoe4 and apoj biomarker-based prevention and treatment of dementia |
US20130209549A1 (en) | 2010-07-21 | 2013-08-15 | University Of South Florida | Materials and methods for treating neurodegenerative diseases |
AU2011335545A1 (en) * | 2010-11-30 | 2013-06-13 | Orphazyme Aps | 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 |
WO2013148333A1 (en) | 2012-03-28 | 2013-10-03 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Salicylic acid derivatives useful as glucocerebrosidase activators |
TW201427695A (zh) * | 2012-11-05 | 2014-07-16 | Genzyme Corp | 用於治療蛋白質病變(proteinopathies)的組成物及方法 |
WO2014079000A1 (en) | 2012-11-21 | 2014-05-30 | Wuhan Yzy Biopharma Co., Ltd. | Bispecific antibody |
CA2938577A1 (en) * | 2014-02-04 | 2015-08-13 | New York University | Progranulin (pgrn) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
PT3193840T (pt) | 2014-09-15 | 2021-08-10 | Orphazyme Aps | Formulação de arimoclomol |
CN107106576A (zh) * | 2014-10-10 | 2017-08-29 | 弗吉尼亚联邦大学 | 用于因减弱的瘦蛋白活性和脂质贮积障碍中的至少一种导致的障碍的疗法的氧化胆固醇硫酸酯类 |
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US11707456B2 (en) | 2020-11-19 | 2023-07-25 | Kempharm Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
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