- METHODS FOR TREATING NEUROLOGICAL SYMPTOMS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
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Methods are provided for treating or preventing neurological symptoms and disorders which are associated with, e.g., lysosomal storage diseases. The methods include enhancing neuronal connectivity within the brain of a subject, increasing brain tissue volume, or preventing or delaying loss of brain tissue volume in a subject. Also provided are methods for monitoring the progression or regression of a neurological disorder, or assessing the onset of a neurological disorder, associated with a lysosomal storage disease, in which brain tissue volume of the subject is measured.
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Page/Page column 92; 97
(2021/08/14)
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- TRICYCLIC DEGRADERS OF IKAROS AND AIOLOS
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Tricyclic cereblon binders for the degradation of Ikaros or Aiolos by the ubiquitin proteasome pathway for therapeutic applications are described.
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Page/Page column 610-611; 617
(2020/10/21)
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- TREATMENT OF CILIOPATHIES USING INHIBITORS OF GLUCOSYLCERAMIDE SYNTHASE (GCS)
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This disclosure relates to a method of treating a ciliopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said method.
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Page/Page column 70-71; 75
(2020/08/22)
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- METHODS FOR TREATING SYMPTOMS AND DISORDERS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
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This disclosure to methods for treating or preventing particular symptoms and disorders which are associated with lysosomal storage diseases using quinuclidine compounds of formula (I), optionally in combination with enzyme replacement therapy. This includes pain, such as abdominal pain, and dermatological disorders, such as angiokeratoma, in a patient having a disease such as Fabry disease. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods.
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Page/Page column 57-59; 62
(2020/08/22)
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- 4,5-diphenyl isoxazole derivative as well as preparation method and application thereof
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The invention discloses a 4,5-diphenyl isoxazole derivative as well as a preparation method and application thereof. Specifically, the invention relates to a 4,5-diphenyl isoxazole derivative of a structure represented by the formula (I), a stereoisomer or pharmaceutically acceptable salt of the derivative, wherein the definition of each substituent in the formula (I) is the same as that in a specification. Compounds of novel structures have the activity of inhibiting heat shock proteins HSP90, can be used for treating cancers, neurodegenerative diseases, inflammatory diseases, autoimmune diseases and ischemic brain injuries, and has wide application prospect.
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Paragraph 0097; 0118; 0119; 0120
(2017/06/02)
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- General and cost-effective synthesis of 1-heteroaryl/arylcycloalkylamines and their broad applications
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A general and cost-effective route has been developed to synthesize 1-heteroarylsubstituted cycloalkylamines from readily available heteroarylacetate in good yields. This synthesis features a LHMDS promoted cyclization and one-pot hydrolysis/Curtius rearr
- Zhang, Dehui,Zheng, Hongchao,Wang, Xiaodong
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p. 1941 - 1953
(2016/04/05)
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- METHODS FOR TREATING PROTEINOPATHIES
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This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.
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Page/Page column 64; 65; 70; 71
(2016/09/26)
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- Glucosylceramide synthase inhibitors
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The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
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Page/Page column 49; 87
(2015/09/28)
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- NOVEL COMPOUNDS USEFUL FOR THE TREATMENT OF DEGENERATIVE AND INFLAMMATORY DISEASES
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Novel [1,2,4]triazolo[1,5-a]pyridine compounds are disclosed that have a Formula represented by the Formula (I). These compounds may be prepared as a pharmaceutical composition, and may be used for the prevention and treatment of a variety of conditions in mammals including humans, including by way of non-limiting example, diseases involving cartilage degradation, bone and/or joint degradation, for example osteoarthritis; and/or conditions involving inflammation or immune responses, such as Crohn's disease, rheumatoid arthritis, psoriasis, allergic airways disease (e.g. asthma, rhinitis), juvenile idiopathic arthritis, colitis, inflammatory bowel diseases, endotoxin-driven disease states (e.g. complications after bypass surgery or chronic endotox in states contributing to e.g. chronic cardiac failure), diseases involving impairment of cartilage turnover (e.g. diseases involving the anabolic stimulation of chondrocytes), congenital cartilage malformations, diseases associated with hypersecretion of IL6 and transplantation rejection (e.g. organ transplant rejection) and proliferative diseases.
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Page/Page column 70
(2010/04/03)
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- Primary 1-Arylcyclopropylamines from aryl cyanides with diethylzinc and titanium alkoxides
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(Matrix presented) 1-Aryl-substituted primary cyclopropylamines are conveniently prepared from aromatic nitriles and diethylzinc. The yields range from 40 to 56% for donor-substituted (five examples) to 62-82% for non- and acceptor-substituted substrates
- Wiedemann, Stefan,Frank, Daniel,Winsel, Harald,De Meijere, Armin
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p. 753 - 755
(2007/10/03)
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- Compounds having activity as inhibitors of cytochrome P450RAI
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Compounds having Formula 2 wherein the symbols have the meaning defined in the specification are inhibitors of the cytochrome P450RAI (retinoic acid inducible) enzyme, and are used for treating diseases responsive to treatment by retinoids.
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Page column 111-112
(2010/01/31)
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- Compounds having activity as inhibitors of cytochrome P450RAI
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Compounds having Formula 1 wherein the symbols have the meaning defined in the specification are inhibitors of the cytochrome P450RAI (retinoic acid inducible) enzyme, and are used for treating diseases responsive to treatment by retinoids.
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Page column 114
(2010/01/31)
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- Compounds having activity as inhibitors of cytochrome P450RAI
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Compounds having Formula 8 wherein the symbols have the meaning defined in the specification are inhibitors of the cytochrome P450RAI (retinoic acid inducible) enzyme, and are used for treating diseases responsive to treatment by retinoids.
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Page column 42, 112-113
(2010/11/29)
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- Compounds having activity as inhibitors of cytochrome P450RAI
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Compounds having Formula 1 wherein the symbols have the meaning defined in the specification are inhibitors of the cytochrome P450RAI (retinoic acid inducible) enzyme, and are used for treating diseases responsive to treatment by retinoids.
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- Methods of providing and using compounds having activity as inhibitors of cytochrome P450RAI
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Novel compounds having the Formulas 1 through 8, wherein the symbols have the meaning defined in the specification, and certain previously known compounds have been discovered to act as inhibitors of the cytochrome P450RAI (retinoic acid inducible) enzyme, and are used for treating diseases responsive to treatment by retinoids. The compound can also be used in co-treatment with retinoids.
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