78742-98-0Relevant academic research and scientific papers
METHODS FOR TREATING NEUROLOGICAL SYMPTOMS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
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Page/Page column 94, (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 71-72, (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 59, (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.
DEUTERATED COMPOUNDS FOR TREATING FABRY, GAUCHER, PARKINSON'S AND RELATED DISEASES AND CONDITIONS, AND COMPOSITIONS AND METHODS THEREOF
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Paragraph 0062; 0063, (2019/08/26)
Novel deuterium-containing chemical compounds are provided. They are glucosylceramide (GSC) synthase inhibitors and are useful for treating various types of lysosomal storage diseases including Fabry's disease, Gaucher's disease and Parkinson's disease, or related diseases and conditions. Also described are pharmaceutical compositions, and methods of preparation and use thereof.
METHOD OF PREPARING GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
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Paragraph 0101, (2017/12/27)
The invention relates to a method of preparing 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.
METHODS FOR TREATING PROTEINOPATHIES
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Page/Page column 66, (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 92, (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.
Process Development of a GCS Inhibitor Including Demonstration of Lossen Rearrangement on Kilogram Scale
Zhao, Jin,Gimi, Rayomand,Katti, Sanjeev,Reardon, Michael,Nivorozhkin, Vitaly,Konowicz, Paul,Lee, Edward,Sole, Lynne,Green, Jerome,Siegel, Craig S.
, p. 576 - 581 (2015/05/27)
A small molecule was under investigation as an inhibitor of glucosylceramide synthase (GCS) for potential use in Fabry disease. To support preclinical activities, a four-step synthesis was developed and used to prepared kilogram quantities of the drug substance. The new route features a scalable CDI-mediated Lossen rearrangement as a substitution for hazardous azide chemistry that was employed in the original route.
METHOD OF PREPARING GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
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Page/Page column 21, (2014/10/04)
The invention relates to a method of preparing 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.
