Advances in Pompe™ Disease: Opportunities to Improve Prognosis Through Early Detection and New Treatments

Target Audience

This online educational activity is directed toward pediatricians and medical specialists who have the opportunity to recognize Pompe disease such as hematologists, oncologists, cardiologists, neurologists, growth specialists, pulmonologists, gastroenterologists, hepatologists, geneticists, and ear, nose, and throat specialists. Additionally, nurse practitioners (NPs), physician assistants (PAs), nurses and other health care professionals who are involved in the diagnosis and management of Pompe disease will be invited to participate.

Program Overview

This online educational activity is designed to provide expert interpretation of new and emerging data on the treatment of Pompe disease, with a forward-looking perspective on how new and novel agents might impact clinical practice. The essentials of diagnosis, current and emerging treatment strategies, and improvements in multispecialty management for Pompe disease will be presented in a multimedia format, including a series of video interviews with expert thought leaders integrated into text-based elements.

Learning Objectives

Upon completion of this activity, participants should be better able to:

Recognize the genetic etiology and known pathophysiological mechanisms involved in Pompe disease and differentiate between phenotypic disease classifications
Identify the clinical presentation of Pompe disease, including the variability and unpredictability of symptoms in some patients, and the importance of early diagnosis
Evaluate the efficacy and safety of investigational enzyme replacement therapy (ERT) for patients with Pompe disease