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Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making

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Target Audience

This educational activity is directed toward neuromuscular specialists who treat patients with Pompe disease. Cardiologists, clinical geneticists, pulmonologists, nurse practitioners, physician assistants, and other health care professionals interested in the treatment of Pompe disease are invited to participate.

Program Overview

Pompe disease is a genetic lysosomal storage disease—the first of these conditions to be identified—that is caused by deficiency of the enzyme acid α-glucosidase. This deficiency causes cellular accumulation of glycogen, resulting in cellular dysfunction, progressive muscle damage and debilitation, and multisystem failure. The rarity and heterogeneity of Pompe disease frequently results in delays in diagnosis of months to years.

This online, on-demand virtual symposium brings together experts in Pompe disease. In this educational program, these experts present key insights to help learners promptly diagnose affected patients and improve their disease management as part of a multidisciplinary approach. This presentation also highlights emerging treatments.

This educational activity is an archive of the live symposium held on September 24, 2022.

Learning Objectives

Upon completion of this activity, participants should be better able to:
  • Apply current evidence-based recommendations to screen and diagnose patients with suspected Pompe disease
  • Evaluate clinical trial data for current and investigational therapies for patients with Pompe disease
  • Design multidisciplinary treatment plans for patients with Pompe disease

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