Kevin Vaughan, associate professor of biology, and affiliated member of the Center for Stem Cells and Regenerative Medicine
Rare neurological diseases represent a unique challenge for scientists because neurons are notoriously difficult to study, and our understanding of basic neurobiology is incomplete. Using Niemann Pick Type C (NPC) disease as an example, universal mechanisms of neuronal loss can be revealed that suggest fundamentally-new strategies to curing disease. These new insights provide hope for a panel of lysosomal storage diseases that lack an effective therapy or cure.
Originally published at science.nd.edu.