Pyrrolopyrimidine compounds and uses thereof for modulating glucocerebrosidase activity
AssigneeNorthwestern University
InventorsDimitri Krainc, Richard B. Silverman, Jianbin Zheng
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AssigneeNorthwestern University
InventorsDimitri Krainc, Richard B. Silverman, Jianbin Zheng
Estimating the term…
Disclosed are new small molecules having a pyrrolopyrimidine core structure and the uses thereof for modulating glucocerebrosidase activity. Also disclosed are pharmaceutical compositions comprising the small molecules which may be administered in methods of treating diseases or disorders associated with glucocerebrosidase activity, including neurological diseases and disorders such as Gaucher's disease and Parkinson's disease. The small molecules may be utilized to generate activated glucocerebrosidase. The activated glucocerebrosidase thusly generated can be administered in enzyme replacement therapy and/or utilized in screening assays for new small molecules that bind to the activated glucocerebrosidase and/or modulate the activity of the activated glucocerebrosidase.
A compound or a pharmaceutically acceptable salt thereof having a Formula I:
A compound or a pharmaceutically acceptable salt thereof having a Formula I:
A compound or a pharmaceutically acceptable salt thereof having a Formula I:
Dependencies are inferred from the claim text.