17797-12-5Relevant academic research and scientific papers
METHODS FOR TREATING NEUROLOGICAL SYMPTOMS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
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Page/Page column 91; 96, (2021/08/14)
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.
TREATMENT OF CILIOPATHIES USING INHIBITORS OF GLUCOSYLCERAMIDE SYNTHASE (GCS)
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Page/Page column 69; 71; 74, (2020/08/22)
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.
METHODS FOR TREATING SYMPTOMS AND DISORDERS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
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Page/Page column 56-57; 59; 62, (2020/08/22)
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.
DIHYDROPYRIMIDINE COMPOUNDS AND USES THEREOF IN MEDICINE
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Paragraph 00217, (2019/01/17)
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.
Dihydropyrimidine compound and uses of dihydropyrimidine compound in drugs
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Paragraph 0534; 0535; 0536; 0537, (2019/01/16)
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.
THIAZOLE DERIVATIVE AND APPLICATIONS THEREOF
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Paragraph 0115; 0116, (2020/01/02)
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.
NITROGEN-CONTAINING SATURATED HETEROCYCLIC COMPOUND
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Paragraph 0344, (2016/08/29)
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].
METHODS FOR TREATING PROTEINOPATHIES
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Page/Page column 63; 64; 70, (2016/09/26)
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.
Glucosylceramide synthase inhibitors
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Page/Page column 48; 51; 86, (2015/09/28)
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.
SUBSTITUTED PYRIMIDINES
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Page/Page column 101, (2013/04/10)
Disclosed are substituted pyrimidines useful as HIF prolyl hydroxylase inhibitors to treat anemia and like conditions.
