MX388562B - 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
- MX388562B MX388562B MX2018012902A MX2018012902A MX388562B MX 388562 B MX388562 B MX 388562B MX 2018012902 A MX2018012902 A MX 2018012902A MX 2018012902 A MX2018012902 A MX 2018012902A MX 388562 B MX388562 B MX 388562B
- Authority
- MX
- Mexico
- Prior art keywords
- lysosomal
- disorders
- methods
- compositions
- treatment
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 208000015439 Lysosomal storage disease Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 230000004064 dysfunction Effects 0.000 title 1
- 230000002132 lysosomal effect Effects 0.000 title 1
- HDTRYLNUVZCQOY-UHFFFAOYSA-N α-D-glucopyranosyl-α-D-glucopyranoside Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(O)C(O)C(CO)O1 HDTRYLNUVZCQOY-UHFFFAOYSA-N 0.000 abstract 1
- HDTRYLNUVZCQOY-WSWWMNSNSA-N Trehalose Natural products O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-WSWWMNSNSA-N 0.000 abstract 1
- HDTRYLNUVZCQOY-LIZSDCNHSA-N alpha,alpha-trehalose Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-LIZSDCNHSA-N 0.000 abstract 1
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)
- 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.
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 MX2018012902A (es) | 2019-06-06 |
| MX388562B true 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014264228A1 (en) | 2013-05-07 | 2015-11-12 | Bio Blast Pharma Ltd. | Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose |
| SI3364958T1 (sl) | 2015-10-23 | 2023-05-31 | Navitor Pharmaceuticals, Inc. | Modulatorji interakcije sestrina in gator2 ter njihova uporaba |
| JP7302871B2 (ja) | 2016-04-21 | 2023-07-04 | ベイラー カレッジ オブ メディスン | リソソーム蓄積障害およびリソソーム機能不全によって特徴付けられる障害を治療するための組成物および方法 |
| 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 |
| CA3117336A1 (en) * | 2018-10-24 | 2020-04-30 | Navitor Pharmaceuticals, Inc. | Polymorphs of (s)-2-amino-5,5-difluoro-4,4-dimethylpentanoic acid for use in modulating mtorc1 |
| CN120714034A (zh) | 2018-12-05 | 2025-09-30 | 华盛顿大学 | 检测、预防、逆转和治疗神经系统疾病的方法 |
| WO2020210798A1 (en) * | 2019-04-12 | 2020-10-15 | Loma Linda University | Methods for treatment of niemann-pick disease type c |
| CA3153963A1 (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 |
| CN114929237A (zh) * | 2019-10-01 | 2022-08-19 | 西洛斯治疗有限公司 | 海藻糖制剂及其用途 |
| IL292612A (en) | 2019-11-01 | 2022-07-01 | Navitor Pharm Inc | Treatment methods using mtorc1 modulator |
| EP4146623A4 (en) | 2020-05-07 | 2024-05-01 | 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 |
| US20250144101A1 (en) * | 2022-01-20 | 2025-05-08 | The Johns Hopkins University | Lysosomal dysfunction in neurological and psychiatric disorders |
| WO2023205415A1 (en) * | 2022-04-22 | 2023-10-26 | Mitobridge Inc. | Transcription factor eb activators and uses thereof |
| 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| ES2779006T3 (es) | 2013-01-09 | 2020-08-13 | Amicus Therapeutics Inc | Composiciones de DNJ parenterales estables |
| US9084720B2 (en) | 2013-05-07 | 2015-07-21 | BioBlast Pharma Ltd. | Compositions and methods for treating oculopharyngeal muscular dystrophy |
| AU2014264228A1 (en) | 2013-05-07 | 2015-11-12 | 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 |
| CA2933554A1 (en) | 2013-12-12 | 2015-06-18 | The Brigham And Women's Hospital, Inc. | Treating neurodegenerative disease |
| 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 |
| CA3012502A1 (en) | 2016-02-08 | 2017-08-17 | Junaxo, Inc. | Use of trehalose for treatment of neurological diseases |
| JP7302871B2 (ja) | 2016-04-21 | 2023-07-04 | ベイラー カレッジ オブ メディスン | リソソーム蓄積障害およびリソソーム機能不全によって特徴付けられる障害を治療するための組成物および方法 |
| 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 |
-
2017
- 2017-04-21 JP JP2019531851A patent/JP7302871B2/ja active Active
- 2017-04-21 CN CN201780038419.8A patent/CN109789316B/zh active Active
- 2017-04-21 US US15/494,136 patent/US10512656B2/en active Active
- 2017-04-21 AU AU2017252563A patent/AU2017252563B2/en active Active
- 2017-04-21 EP EP17786739.7A patent/EP3445451B1/en active Active
- 2017-04-21 NZ NZ748599A patent/NZ748599A/en unknown
- 2017-04-21 DK DK17786739.7T patent/DK3445451T3/da active
- 2017-04-21 MX MX2018012902A patent/MX388562B/es unknown
- 2017-04-21 WO PCT/US2017/028904 patent/WO2017185010A1/en not_active Ceased
-
2019
- 2019-04-04 US US16/375,445 patent/US11083741B2/en active Active
- 2019-04-04 US US16/375,521 patent/US20200000832A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP3445451A4 (en) | 2019-12-18 |
| BR112018071684A2 (pt) | 2019-02-19 |
| JP7302871B2 (ja) | 2023-07-04 |
| CA3021846A1 (en) | 2017-10-26 |
| AU2017252563B2 (en) | 2022-07-21 |
| NZ748599A (en) | 2022-08-26 |
| US20200000832A1 (en) | 2020-01-02 |
| US20170304339A1 (en) | 2017-10-26 |
| MX2018012902A (es) | 2019-06-06 |
| JP2021517552A (ja) | 2021-07-26 |
| WO2017185010A1 (en) | 2017-10-26 |
| US11083741B2 (en) | 2021-08-10 |
| US10512656B2 (en) | 2019-12-24 |
| US20190321383A1 (en) | 2019-10-24 |
| AU2017252563A1 (en) | 2018-12-06 |
| CN109789316A (zh) | 2019-05-21 |
| DK3445451T3 (da) | 2021-11-08 |
| EP3445451A1 (en) | 2019-02-27 |
| EP3445451B1 (en) | 2021-08-18 |
| CN109789316B (zh) | 2022-09-27 |
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