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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.

Info

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.)
Filing date
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

Links

Classifications

    • 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
    • A61K31/7016Disaccharides, e.g. lactose, lactulose
    • 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
    • A61K31/4375Heterocyclic 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
    • 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
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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

Landscapes

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

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201509030TA (en) 2013-05-07 2015-11-27 Bio Blast Pharma Ltd Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
ES2941969T3 (es) 2015-10-23 2023-05-29 Navitor Pharm Inc Moduladores de la 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
EP4556069A3 (en) 2017-04-26 2025-08-13 Navitor Pharmaceuticals, Inc. Modulators of sestrin-gator2 interaction and uses thereof
JP7644002B2 (ja) * 2018-10-24 2025-03-11 ナビター ファーマシューティカルズ, インコーポレイテッド 多形化合物およびその使用
US12460262B2 (en) 2018-12-05 2025-11-04 Washington University Methods of detecting, preventing, reversing, and treating neurological diseases
WO2020210798A1 (en) * 2019-04-12 2020-10-15 Loma Linda University Methods for treatment of niemann-pick disease type c
WO2021050102A1 (en) * 2019-09-09 2021-03-18 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
IL291799A (en) * 2019-10-01 2022-06-01 Seelos Therapeutics Inc Trehalose formulations and uses thereof
TWI870497B (zh) 2019-11-01 2025-01-21 美商奈維特製藥公司 使用mtorc1調節劑的治療方法
AU2021269231B2 (en) 2020-05-07 2025-11-13 Alectos Therapeutics Inc. Non-lysosomal glucosylceramidase inhibitors and uses thereof
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
EP4465990A2 (en) * 2022-01-20 2024-11-27 The Johns Hopkins University Lysosomal dysfunction in neurological and psychiatric disorders
CA3256283A1 (en) * 2022-04-22 2023-10-26 Astellas Engineered Small Molecules US, Incorporated EB TRANSCRIPTION FACTOR ACTIVATORS AND THEIR USES
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

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69529026T2 (de) * 1994-07-19 2003-07-17 Kabushiki Kaisha Hayashibara Seibutsu Kagaku Kenkyujo, Okayama Trehalose, ihre Herstellung und ihre Verwendung
CA2612538C (en) 2005-05-17 2015-06-30 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
ES2758827T3 (es) * 2009-06-17 2020-05-06 Biomarin Pharm Inc Formulaciones para enzimas lisosómicas
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
CN104918619B (zh) 2013-01-09 2019-04-02 阿米库斯治疗学公司 稳定的肠胃外dnj组合物
US9084720B2 (en) 2013-05-07 2015-07-21 BioBlast Pharma Ltd. Compositions and methods for treating oculopharyngeal muscular dystrophy
SG11201509030TA (en) 2013-05-07 2015-11-27 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
WO2014200705A1 (en) * 2013-06-14 2014-12-18 Stc.Unm Treatment of autophagy-related disorders
CN106030302B (zh) 2013-12-12 2019-09-24 布里格姆妇女医院 治疗神经变性疾病
ES2732308T3 (es) * 2013-12-23 2019-11-21 Bcn Peptides Sa Análogos de bicalutamida o (s)-bicalutamida como compuestos activadores de la exocitosis para su uso en el tratamiento de un desorden de acúmulo lisosomal o glucogenosis
EP3413898A4 (en) 2016-02-08 2019-09-25 Junaxo, Inc. USE OF TREHALOSIS TO TREAT NEUROLOGICAL ILLNESSES
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
BR112018071684A2 (pt) 2019-02-19
EP3445451A4 (en) 2019-12-18
MX388562B (es) 2025-03-20
US11083741B2 (en) 2021-08-10
EP3445451B1 (en) 2021-08-18
US20170304339A1 (en) 2017-10-26
CN109789316B (zh) 2022-09-27
NZ748599A (en) 2022-08-26
CN109789316A (zh) 2019-05-21
US20190321383A1 (en) 2019-10-24
WO2017185010A1 (en) 2017-10-26
US10512656B2 (en) 2019-12-24
DK3445451T3 (da) 2021-11-08
EP3445451A1 (en) 2019-02-27
JP2021517552A (ja) 2021-07-26
AU2017252563A1 (en) 2018-12-06
AU2017252563B2 (en) 2022-07-21
CA3021846A1 (en) 2017-10-26
JP7302871B2 (ja) 2023-07-04
US20200000832A1 (en) 2020-01-02

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