Pharmaceutical formulations comprising substituted pyrazolo[5,1-c]pyrido[4,3-e][1,2,4]triazines for treating lysosomal storage disorders
Abstract:
This disclosure provides compounds, such as compounds of Formula I: pharmaceutical formulations thereof, and related methods of use for the treatment of defects of cholesterol homeostasis including lysosomal storage disorders such as Niemann-Pick Type C disease.
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