Enhancing Recognition of Late‐Onset Pompe Disease to Facilitate Personalized, Multidisciplinary Care and Maintain Function: The Promise of Emerging Agents

TARGET AUDIENCE:
The educational design of this activity addresses the needs of neurologists (adult and pediatric), internal medicine physicians, primary care physicians, pediatricians, pharmacists, neurology advanced practice providers and nurses, and other clinicians who care for patients with LOPD.
PROGRAM OVERVIEW:
Patients with late‐onset Pompe disease (LOPD) often experience significant diagnostic delays and are susceptible to potential misdiagnoses. In addition, current treatments are associated with a number of limitations, leading to investigations of new agents that may offer greater safety, tolerability, and efficacy.

Join Dr. Loren Peña to review the symptoms of LOPD, patient factors that might influence care, and advances in the management of LOPD. Dr. Peña will discuss how to effectively incorporate new treatments, when available, into daily clinical practice and differentiate current and emerging agents to determine their appropriate place in the care of LOPD. Clinicians will be prepared to engage their patients in effective shared decision-marking to develop care plans that address the needs of their patients with LOPD.
LEARNING OBJECTIVES:
EDUCATIONAL OBJECTIVES
After completing this activity, the participant should be better able to:
• Recognize signs and symptoms that may raise the index of suspicion for LOPD
• Identify strategies and emerging agents to address limitations of current treatment options for LOPD
• Assess the comparative safety, efficacy, and tolerability profiles and therapeutic potential of agents being investigated for the treatment of LOPD
• Discuss the importance of engaging patients with LOPD via shared decision‐making and how it could impact outcomes