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Description

Despite the proven benefits of enzyme replacement therapy (ERT) for patients with Pompe disease (PD), many individuals continue to experience disease progression while on therapy. Next-generation ERT and combination ERT/enzyme stabilizer therapy have been developed to address this limitation of conventional ERT. This activity will explore the most recent data on the latest advances for the treatment of PD and evaluate practical strategies that may guide the practice of users of newer therapies as they become available.

TARGET AUDIENCE
This initiative is intended for neuromuscular specialists, neurologists, clinical geneticists, genetic counselors, inherited metabolic disease/lysosomal storage disorder specialists, physical medicine and rehabilitation clinicians, orthopedists, pulmonologists, pediatricians, primary care/family medicine clinicians, as well as cardiologists, hepatologists, and other healthcare providers involved in the diagnosis and management of PD.

AGENDA
• Unmet Needs Among Individuals With PD
• Exploring Next-Generation ERT Formulas in Addressing Unmet PD Needs
• Tailoring Therapy to Disease Progression—Featuring a Clinical Case Challenge

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

• Recognize unmet diagnostic and treatment needs of patients with PD
• Assess the efficacy and safety of next-generation ERT and ERT/enzyme stabilizer formulas for PD, including how they compare with first-generation therapy
• Evaluate methods to assess and monitor PD severity and progression to inform treatment decision-making
• Identify PD clinical scenarios in which an ERT/enzyme stabilizer or second-generation ERT is likely to be beneficial

FACULTY
Priya Kishnani, MD, MBBS (Course Director)
Duke University Medical Center
Durham, NC

Professor Dr. med. Benedikt Schoser, FEAN
Ludwig-Maximilians-University Munich 
Munich, Germany

Prof. Antonio Toscano
University of Messina
Messina, Italy

Criteria for Success
Certificates of completion will be awarded based on the participant’s attendance and submission of the activity evaluation/claim credit form. You must participate in the entire activity to receive credit. There is no fee to participate in this activity. If you have questions about this activity, please contact AKH Inc. at tbrignoni@akhcme.com.

Credit provided by AKH Inc., Advancing Knowledge in Healthcare

In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC. AKH Inc., Advancing Knowledge in Healthcare is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team

Physicians
AKH Inc., Advancing Knowledge in Healthcare designates this live activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

This activity is jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC.

This activity is supported by educational grant from Amicus Therapeutics, Inc.