MX2018012902A - Composiciones y métodos para el tratamiento de trastornos de almacenamiento lisosomal y trastornos caracterizados por disfunción lisosomal. - Google Patents

Composiciones y métodos para el tratamiento de trastornos de almacenamiento lisosomal y trastornos caracterizados por disfunción lisosomal.

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Publication number
MX2018012902A
MX2018012902A MX2018012902A MX2018012902A MX2018012902A MX 2018012902 A MX2018012902 A MX 2018012902A MX 2018012902 A MX2018012902 A MX 2018012902A MX 2018012902 A MX2018012902 A MX 2018012902A MX 2018012902 A MX2018012902 A MX 2018012902A
Authority
MX
Mexico
Prior art keywords
lysosomal
disorders
methods
compositions
treatment
Prior art date
Application number
MX2018012902A
Other languages
English (en)
Other versions
MX388562B (es
Inventor
Sardiello Marco
Original Assignee
Baylor College Medicine
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.)
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Publication date
Application filed by Baylor College Medicine filed Critical Baylor College Medicine
Publication of MX2018012902A publication Critical patent/MX2018012902A/es
Publication of MX388562B publication Critical patent/MX388562B/es

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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
    • A61K31/7016—Disaccharides, e.g. lactose, lactulose
    • 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
    • A61K31/4353—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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4375—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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención se refiere a composiciones y métodos para tratar enfermedades de almacenamiento lisosomal y métodos para usar trehalosa.
MX2018012902A 2016-04-21 2017-04-21 Composiciones y métodos para el tratamiento de trastornos de almacenamiento lisosomal y trastornos caracterizados por disfunción lisosomal. MX388562B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662325535P 2016-04-21 2016-04-21
US201762475295P 2017-03-23 2017-03-23
PCT/US2017/028904 WO2017185010A1 (en) 2016-04-21 2017-04-21 Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction

Publications (2)

Publication Number Publication Date
MX2018012902A true MX2018012902A (es) 2019-06-06
MX388562B MX388562B (es) 2025-03-20

Family

ID=60089338

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018012902A MX388562B (es) 2016-04-21 2017-04-21 Composiciones y métodos para el tratamiento de trastornos de almacenamiento lisosomal y trastornos caracterizados por disfunción lisosomal.

Country Status (9)

Country Link
US (3) US10512656B2 (es)
EP (1) EP3445451B1 (es)
JP (1) JP7302871B2 (es)
CN (1) CN109789316B (es)
AU (1) AU2017252563B2 (es)
DK (1) DK3445451T3 (es)
MX (1) MX388562B (es)
NZ (1) NZ748599A (es)
WO (1) WO2017185010A1 (es)

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WO2014181333A2 (en) 2013-05-07 2014-11-13 Bio Blast Pharma Ltd. Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
MX389001B (es) 2015-10-23 2025-03-20 Navitor Pharm Inc Moduladores de interacción de sestrina-gator2 y sus usos.
US10512656B2 (en) 2016-04-21 2019-12-24 Baylor College Of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
ES3040534T3 (en) 2017-04-26 2025-11-03 Navitor Pharm Inc Modulator of sestrin-gator2 interaction for use in the treatment of treatment-resistant depression
WO2020086816A1 (en) * 2018-10-24 2020-04-30 Navitor Pharmaceuticals, Inc. Polymorphic compounds and uses thereof
KR20210113606A (ko) 2018-12-05 2021-09-16 워싱턴 유니버시티 신경 질환을 검출, 예방, 회복 및 치료하는 방법
US20220211813A1 (en) * 2019-04-12 2022-07-07 Loma Linda University Methods for treatment of niemann-pick disease type c
US12612380B2 (en) 2019-05-10 2026-04-28 Alectos Therapeutics 1Nc Non-lysosomal glucosylceramidase inhibitors and uses thereof
US20220313722A1 (en) * 2019-09-09 2022-10-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
CA3150309A1 (en) 2019-10-01 2021-04-08 Seelos Therapeutics, Inc. Trehalose formulations and uses thereof
AU2020376950B2 (en) 2019-11-01 2026-06-11 Supernus Pharmaceuticals, Inc. Methods of treatment using an mTORC1 modulator
PE20231423A1 (es) 2020-05-07 2023-09-13 Alectos Therapeutics Inc Inhibidores de glucosilceramidasa no lisosomal y los usos de los mismos
EP3944858A1 (en) 2020-07-30 2022-02-02 Beyond Batten Disease Foundation Combination product containing trehalose and miglustat for treating lysosomal diseases
US12019081B2 (en) 2020-11-06 2024-06-25 University Of Kentucky Research Foundation Method for detection and quantification of CLN3 protein
KR102580852B1 (ko) * 2021-04-13 2023-09-20 강원대학교 산학협력단 발리다마이신 a를 포함하는 뇌전증 완화 및 치료용 조성물
US20250144101A1 (en) * 2022-01-20 2025-05-08 The Johns Hopkins University Lysosomal dysfunction in neurological and psychiatric disorders
TW202400160A (zh) * 2022-04-22 2024-01-01 美商米突倍基公司 轉錄因子eb活化子及其用途
CN116819066A (zh) * 2023-06-30 2023-09-29 复旦大学附属中山医院 肝癌微环境检测试剂盒
US12639967B2 (en) 2023-07-05 2026-05-26 Nice North America Llc Systems and methods for detecting and recognizing a railcar identifier

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EP0693558B1 (en) * 1994-07-19 2002-12-04 Kabushiki Kaisha Hayashibara Seibutsu Kagaku Kenkyujo Trehalose and its production and use
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
GB0712494D0 (en) 2007-06-27 2007-08-08 Isis Innovation Substrate reduction therapy
US8785168B2 (en) * 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
US20130312784A1 (en) 2012-05-24 2013-11-28 For Your Ease Only, Inc. Cosmetic organizer
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
AU2014205378B2 (en) 2013-01-09 2018-08-30 Amicus Therapeutics, Inc. Stable parenteral DNJ compositions
US9084720B2 (en) 2013-05-07 2015-07-21 BioBlast Pharma Ltd. Compositions and methods for treating oculopharyngeal muscular dystrophy
WO2014181333A2 (en) 2013-05-07 2014-11-13 Bio Blast Pharma Ltd. Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
JP6130224B2 (ja) * 2013-05-27 2017-05-17 公益財団法人微生物化学研究会 新規化合物レンツトレハロース、その製造方法、及びその用途、並びに、新規微生物
US20160136123A1 (en) * 2013-06-14 2016-05-19 Vojo P. Deretic Treatment of autophagy-related disorders
WO2015089443A2 (en) 2013-12-12 2015-06-18 The Brigham And Women's Hospital, Inc. Treating neurodegenerative disease
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CA3012502A1 (en) * 2016-02-08 2017-08-17 Junaxo, Inc. Use of trehalose for treatment of neurological diseases
US10512656B2 (en) 2016-04-21 2019-12-24 Baylor College Of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
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

Also Published As

Publication number Publication date
AU2017252563B2 (en) 2022-07-21
EP3445451B1 (en) 2021-08-18
US20170304339A1 (en) 2017-10-26
DK3445451T3 (da) 2021-11-08
US20200000832A1 (en) 2020-01-02
CA3021846A1 (en) 2017-10-26
EP3445451A4 (en) 2019-12-18
CN109789316B (zh) 2022-09-27
US11083741B2 (en) 2021-08-10
NZ748599A (en) 2022-08-26
US10512656B2 (en) 2019-12-24
BR112018071684A2 (pt) 2019-02-19
AU2017252563A1 (en) 2018-12-06
JP2021517552A (ja) 2021-07-26
MX388562B (es) 2025-03-20
EP3445451A1 (en) 2019-02-27
WO2017185010A1 (en) 2017-10-26
JP7302871B2 (ja) 2023-07-04
US20190321383A1 (en) 2019-10-24
CN109789316A (zh) 2019-05-21

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