- 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 91; 96
(2021/08/14)
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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 69; 71; 74
(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 56-57; 59; 62
(2020/08/22)
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- DIHYDROPYRIMIDINE COMPOUNDS AND USES THEREOF IN MEDICINE
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Provided herein are a dihydropyrimidine compound and a pharmaceutical application thereof, especially the application used for treating and preventing HBV diseases. Specifically, provided herein is a compound having Formula (I) or (Ia), or an enantiomer, a diastereoisomer, a tautomer, a hydrate, a solvate, or a pharmaceutically acceptable salt thereof, wherein the variables of the formulas are as defined in the specification. Also provided herein is use of the compound having Formula (I) or (Ia), or an enantiomer, a diastereoisomer, a tautomer, a hydrate, a solvate, or a pharmaceutically acceptable salt thereof as a medicine, especially for treating and preventing HBV diseases.
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Paragraph 00217
(2019/01/17)
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- Dihydropyrimidine compound and uses of dihydropyrimidine compound in drugs
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The present invention relates to a dihydropyrimidine compound and uses of the dihydropyrimidine compound as drugs, particularly as drugs for treatment and prevention of hepatitis B, particularly to acompound represented by a general formula (I) or (Ia) or an enantiomer, a diastereomer, a tautomer, a hydrate, a solvate or a pharmaceutically acceptable salt thereof, wherein each variable is definedin the specification. The invention further relates to uses of the compound represented by a general formula (I) or (Ia) or the enantiomer, the diastereomer, the tautomer, the hydrate, the solvate orthe pharmaceutically acceptable salt thereof as drugs, especially as drugs for treatment and prevention of hepatitis B. The formulas (I) and (Ia) are defined in the specification.
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Paragraph 0534; 0535; 0536; 0537
(2019/01/16)
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- THIAZOLE DERIVATIVE AND APPLICATIONS THEREOF
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Disclosed in the present invention are a new thiazole compound, particularly a compound represented by formula (I), a pharmaceutical composition thereof and applications thereof in the preparation of drugs for the treatment of diseases related to herpes simplex viruses.
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Paragraph 0115; 0116
(2020/01/02)
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- NITROGEN-CONTAINING SATURATED HETEROCYCLIC COMPOUND
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The present invention provides a compound represented by the following formula (I) or its pharmaceutically acceptable salt: [wherein, R1 represents optionally substituted C1-4 alkyl, n shows integer of 1 to 4, R2 represents optionally substituted C1-4 alkyl or hydrogen atom, R3 represents optionally substituted C1-4 alkyl, R4a, R4b, R4c, and R4d, similarly or differently, represent optionally substituted C6-14 aryl, optionally substituted C1-4 alkyl, or hydrogen atom and the like, A represents optionally substituted C6-14 aryl or optionally substituted 5 to 11 membered heteroaryl].
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Paragraph 0344
(2016/08/29)
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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 63; 64; 70
(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 48; 51; 86
(2015/09/28)
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- SUBSTITUTED PYRIMIDINES
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Disclosed are substituted pyrimidines useful as HIF prolyl hydroxylase inhibitors to treat anemia and like conditions.
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Page/Page column 101
(2013/04/10)
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- SUBSTITUTED PYRIMIDINES
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The present invention relates to substituted pyrimidines useful as HIF prolyl hydroxylase inhibitors to treat anemia and like conditions.
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Page/Page column 101
(2013/04/10)
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- COMPOUNDS AND COMPOSITIONS AS SYK KINASE INHIBITORS
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Provided herein area novel class of compounds, pharmaceutical compositions comprising such compounds and methods of using such compounds to treat or prevent diseases or disorders associated with abnormal or deregulated Syk kinase activity.
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Page/Page column 85-86
(2011/02/24)
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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 71
(2010/04/03)
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- Pyrrolopyrimidines and Pyrrolopyridines
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Compounds of formula I in free or salt or solvate form, wherein X, T1, T3 and T4 have the meanings as indicated in the specification, are useful for treating diseases mediated by the ALK-5 and/or ALK-4 receptor. Pharmaceutical compositions that contain the compounds and processes for preparing the compounds are also described.
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Page/Page column 54
(2009/07/25)
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- Synthesis and biological study of 2-amino-4-aryl-5-chloropyrimidine analogues as inhibitors of VEGFR-2 and cyclin dependent kinase 1 (CDK1)
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The series of 2-amino-4-aryl-5-chloropyrimidines was discovered to be potent for both VEGFR-2 and CDK1. Described here are the chemistry for analogue synthesis, SAR study, and its kinase selectivity prolifing. The full rat PK data and in vivo efficacy stu
- Huang, Shenlin,Li, Ronghua,Connolly, Peter J.,Emanuel, Stuart,Fuentes-Pesquera, Angel,Adams, Mary,Gruninger, Robert H.,Seraj, Jabed,Middleton, Steven A.,Davis, Jeremy M.,Moffat, David F.C.
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p. 2179 - 2183
(2008/02/01)
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- 4,5-disubstituted-2-aminopyrimidines
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Pyrimidines of formla (1) are described: wherein R1is a —XR6group; R2and R3which may be the same or different is each a hydrogen or halogen atom or a group selected from an optionally substituted aliphatic, cycl
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