Moving Forward in Pompe Disease

ACTIVITY OVERVIEW – There has been significant progress in the treatment paradigms of Pompe disease (PD) in recent years with the approval of a second-generation enzyme replacement therapy (ERT) and another agent undergoing regulatory review. Additionally, growing access to newborn screening (NBS) is allowing for earlier diagnosis, particularly for individuals with late-onset PD (LOPD). As a result, treatment decision-making is becoming more complex. This activity will discuss the latest clinical data on second-generation ERTs and how they may address limitations of first-generation ERT, methods for assessing disease progression and severity to inform management decision-making, and case presentations across the spectrum of PD onset and severity. Also, management considerations relating to the expansion of NBS programs will be explored, particularly as they relate to early diagnosis and early ERT initiation.

TARGET AUDIENCE

This initiative is intended for Neuromuscular specialists, PM&R clinicians, Neurologists, Cardiologists, Genetic Counselors, Geneticists, and other HCPs who manage individuals with PD.

Host or Publisher Catalyst Medical Education, LLC