Exploring Next-generation Therapies to Mitigate Disease Progression in PD

6001 Destination Parkway,Orlando FL 32819

22 February, 2023

Description

ACTIVITY OVERVIEW 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 NeedsTailoring Therapy to Disease Progression—Featuring a Clinical Case ChallengeQ&A and Closing RemarksLEARNING OBJECTIVES Upon successful completion of this activity, participants should be better able to: Recognize unmet diagnostic and treatment needs of patients with PDAssess the efficacy and safety of next-generation ERT and ERT/enzyme stabilizer formulas for PD, including how they compare with first-generation therapyEvaluate methods to assess and monitor PD severity and progression to inform treatment decision-makingIdentify PD clinical scenarios in which an ERT/enzyme stabilizer or second-generation ERT is likely to be beneficialThis activity is jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC. This activity is supported by an educational grant from Amicus Therapeutics Inc. Eligibility: Registration for WORLDSymposium 2023 and an official name badge are required to attend this satellite symposium.