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Updated: May 27, 2025

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Synthesis of a Hydroxy-15-Azasterol

Caleb A H Jones1,2, Bruce J Melancon1,2, Craig W Lindsley1,2,3

  • 1Warren Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, Tennessee 37232, United States.

ACS Omega
|February 17, 2025
PubMed
Summary

Researchers synthesized a novel hydroxy-15-azasterol compound to treat Niemann-Pick type C (NPC) disease, a rare lysosomal storage disorder. This new azasterol shows potential as a cholesterol mimic for NPC therapy.

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