A new era of late-onset Pompe disease (LOPD) disease management is here with the availability of multiple second-generation enzyme replacement therapies. Explore these activities to gain expert knowledge on the latest strategies and tools for monitoring disease progression. Hear expert insights on when and how to switch enzyme therapies. Review resources to help guide clinical decision-making on the frontlines of patient care.
Courses
Homing in on Holistic Management of Late-Onset Pompe Disease: Contemporary Insights on Disease Monitoring, Treatment Selection and Switching, and Shared Decision-Making
Credit Available: 1.00 AMA PRA Category 1 Credit(s)™
A deeper understanding of Pompe disease has revealed a complex, multisystem disorder with a broad spectrum of manifestations, particularly in patients with late-onset Pompe disease (LOPD). Expanded newborn screening programs have further revealed the insidious nature of symptom onset before overt clinical signs appear. Emerging evidence supports the use of advanced imaging techniques — such as muscle ultrasound and magnetic resonance imaging — for detecting early neuromuscular changes, even before abnormalities in creatine kinase or urine hexose tetrasaccharide levels are observed. Join our expert panel for a dynamic, case-based discussion on evolving insights into disease progression and how next-generation therapies are reshaping the management of LOPD. Gain practical strategies for diagnosis, monitoring, treatment selection, and shared decision-making in an era of expanding therapeutic options.
Credit Available
CME (Physicians): 1 AMA PRA Category 1 Credit™
ANCC (Nurses): 1 ANCC Contact Hours
ACPE (Pharmacists): 1 ACPE Contact Hours
IPCE: 1 Interprofessional Continuing Education Credit
CEU: 1 Continuing Education Units
Course Faculty
Identifying Disease Progression and Making Treatment Switching Decisions in LOPD
Credit Available: 0.5 AMA PRA Category 1 Credit(s)™
Join our experts as they discuss a real-world case on how to evaluate the subtle signs and symptoms of disease progression in late-onset Pompe disease (LOPD). Use of traditional biomarkers, such as urine creatine and Hex4, alongside functional testing and patient-reported outcomes are emphasized. Hear how to best incorporate these assessments into routine practice to support timely, individualized treatment decisions to optimize long-term patient outcomes.
Credit Available
CME (Physicians): 0.5 AMA PRA Category 1 Credit™
ANCC (Nurses): 0.5 ANCC Contact Hours
ACPE (Pharmacists): 0.5 ACPE Contact Hours
IPCE: 0.5 Interprofessional Continuing Education Credit
CEU: 0.5 Continuing Education Units
Course Faculty
Establishing Multidisciplinary, Patient-Centered Care for Adults with LOPD
Credit Available: 0.5 AMA PRA Category 1 Credit(s)™
Hear our experts deliberate the complexities of monitoring and managing late-onset Pompe disease (LOPD) over time. Learn how coordinated, multidisciplinary care alongside consistent monitoring of patient reported outcomes and biomarkers can support clinical decision-making. Gain insights on when and how to switch enzyme replacement therapies to enhance long-term health and functional outcomes for patients.
Credit Available
CME (Physicians): 0.5 AMA PRA Category 1 Credit™
ANCC (Nurses): 0.5 ANCC Contact Hours
ACPE (Pharmacists): 0.5 ACPE Contact Hours
IPCE: 0.5 Interprofessional Continuing Education Credit
CEU: 0.5 Continuing Education Units
Course Faculty
Previous Course
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.
[Accreditation has expired. For informational purposes only.]
Course Faculty
Priya S. Kishnani, MD (Course Director)
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.
[Accreditation has expired. For informational purposes only.]
Course Faculty
Priya S. Kishnani, MD (Course Director)
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)