Survival Analysis of Late-Onset Pompe Disease through Cox Proportional Hazards by Will Singleton ’24

Survival Analysis of Late-Onset Pompe Disease through Cox Proportional Hazards by Will Singleton ’24, Wednesday February 7th, 1:10 – 1:50pm, North Science Building 015, Wachenheim

Abstract:

Pompe Disease is a rare genetic disorder that often results in a lifespan of less than a year. When diagnosed after the age of one (late-onset Pompe disease), it causes muscle weakness, respiratory issues, and a reduced lifespan. A key question is whether the use of treatments such as enzyme replacement therapy (ERT) delays the time until critical symptom onset or death. We will outline common methods for analysis of time-to-event data, including Kaplan-Meier survival curves and the Cox proportional hazards model, then apply them to explore the effects of ERT treatment in late-onset Pompe disease.