Advancing Patient Care in Late-Onset Pompe Disease: Utilizing Patient Perspectives to Facilitate Timely Diagnosis and Patient-Centered Therapy

Diagnosis and management of late-onset Pompe disease (LOPD) involves a variety of complex clinical processes and decisions. Even as newborn screening (NBS) becomes more prevalent, unidentified patients remain, often with vague symptoms that can lead to lengthy diagnostic odysseys. Once patients are identified, clinicians must navigate an evolving debate regarding when to initiate enzyme replacement therapy (ERT) as well as how best to manage patients receiving ERT over the long term in the evolving treatment landscape. This discussion-based activity is designed to address these complexities via expert review of modern literature and the integration of patient and caregiver insights. To inform the program s development, the faculty and organizers collaborated with 15 individuals with LOPD and/or their caregivers to better understand how their diagnostic and treatment journeys shaped their expectations, highlighted unmet needs, and revealed opportunities to strengthen clinician-patient communication and care planning. The resultant program is intended to help learners refine their clinical approaches, engage more effectively in shared decision-making (SDM), and improve outcomes for those living with LOPD.

Credit & MOC Details

Credit information is provided by the course provider. We recommend confirming credit eligibility with the provider or your licensing board before starting the activity.

Target Specialties

Pricing

Free

Reported to ACCME by the provider

Enduring / On-demand
Credit · Up to 1 credit
Commercial Support

Released

Nov 28, 2025
Last day to claim credit

Nov 29, 2026 7 weeks left

Advancing Patient Care in Late-Onset Pompe Disease: Utilizing Patient Perspectives to Facilitate Timely Diagnosis and Patient-Centered Therapy

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