Niemann-Pick Disease Type C

Cytokinetics Announces Positive Results From SEQUOIA-HCM, the Pivotal Phase III Clinical Trial of Aficamten in Patients With Obstructive Hypertrophic Cardiomyopathy Cytokinetics, Incorporated released favorable top-line findings from the SEQUOIA-HCM trial (Safety, Efficacy, and Quantitative Understanding of Obst...

Niemann-Pick disease type C (NPC) is a rare genetic disorder caused by mutations in cholesterol trafficking proteins. There are two main types, with Type C1 being the most prevalent, accounting for around 95% of cases. While specific epidemiological data on NPC is limited, estimates suggest a prevalence of approxim...