This website includes freely accessible education for healthcare providers involved in the diagnosis and management of Pompe disease (PD). Please visit our resources page to access additional information, infographics, and suggested readings.

Exploring Next-Generation Therapies to Mitigate Disease Progression in

POMPE DISEASE:

Exploring Next-Generation ERT Formulas in Addressing Unmet Pompe Disease Needs.

Courses

Optimizing Outcomes in Late-Onset Pompe Disease: Integrating New Therapies, Whole-Person Markers for Disease Monitoring, and Shared Decision-making Into Practice

Credit Available: 1.00 AMA PRA Category 1 Credit(s)™

This activity will explore the new understanding around LOPD symptom onset and the efficacy of next-generation treatments to modify disease progression presented at the 2024 World Muscle Society Congress. Case-based scenarios that explore treatment initiation, how to monitor disease progression with the emerging biomarkers and patient-reported outcomes, strategies for multidisciplinary care that include shared-decision making, and considerations around treatment switching will be discussed.

Course Faculty

Priya S. Kishnani, MD (Course Director)

Mark Roberts, BSc, MBChB, FRCP, MD

Professor Dr. med. Benedikt Schoser, FEAN

Exploring Next-generation Therapies to Mitigate Disease Progression in Pompe Disease

Credit Available: .75 AMA PRA Category 1 Credit(s)™

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 conventionalERT. 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.

Course Faculty

Priya S. Kishnani, MD (Course Director)

Professor Dr. med. Benedikt Schoser, FEAN

Antonio Toscano, MD

Previous Course

Addressing Unmet Needs in Pompe Disease: The Role of New Treatments in Mitigating Disease Progression

Credit Available: 1.25 AMA PRA Category 1 Credit(s)™

This activity will present data from selected abstracts on Pompe Disease (PD) presented at the 2021 World Muscle Society Congress. Focusing on unmet needs and emerging therapies, this dynamic, roundtable style format, will review study design and results, as well as evaluate their potential implications on the treatment of PD in clinical practice.

[Accreditation has expired. For informational purposes only.]

Course Faculty

Priya S. Kishnani, MD (Course Director)

Professor Dr. med. Benedikt Schoser, FEAN

Antonio Toscano, MD