Endpoint Considerations to Facilitate Drug Developmentfor Niemann-Pick Type C (NPC) Day 2

Niemann-Pick Type C (NPC) is a rare genetic disease that results in progressive neurological symptoms
and organ dysfunction. NPC is caused by mutations in either the NPC1 or NPC2 genes, resulting in
impaired intracellular transport of cholesterol and other lipids. Individuals with NPC have significant
unmet treatment needs. Currently, there are no approved therapies in the United States for treatment
of NPC. In order to advance NPC drug development, it is important that stakeholders work together and
identify strategies to support ongoing and future NPC clinical trials. In this workshop, participants will
discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic
development for patients with NPC.