Transforming the Course of Pompe: Advancements in the Care of Evolving Phenotypes for Patients With Late-Onset Disease

Program Description

Newborn screening (NBS) for Pompe disease (PD) enables earlier diagnosis and life-saving treatments, revealing a higher prevalence than previously known. This increases clinician awareness, crucial for those without NBS access, and extends life expectancy, especially for late-onset PD (LOPD), raising new long-term care questions.

While infantile-onset PD (IOPD) receives prompt treatment, LOPD patients face an unpredictable asymptomatic phase and unclear treatment guidelines. The multi-systemic nature of LOPD, revealed by extended life with enzyme replacement therapy (ERT), adds complexity. As therapies improve, it's vital to determine when to start treatment and monitor disease progression.

This activity allows learners to compare their practices with international experts, helping them reflect on their knowledge, identify deviations from best practices, and develop evidence-based strategies for optimal clinical decisions.

Target Audience

This activity is intended for pediatric and adult neurologists, geneticists, pediatricians, physician assistants, and other healthcare professionals involved in the diagnosis and long-term management of patients with Pompe disease.

Learning Objectives

Upon completion of the educational activity, participants should be able to:

  • Identify optimal diagnostic algorithms with or without newborn screening (NBS) to obtain an early and accurate diagnosis in patients with late-onset Pompe disease (LOPD)
  • Evaluate multisystemic signs and symptoms of LOPD progression to effectively monitor patients, promptly initiate enzyme replacement therapy (ERT), and respond to disease worsening
  • Appraise mechanisms of action and phase 3 data of previously available and recently approved next generation ERTs to identify how and for whom these approaches may be integrated into current practice
  • Describe the biologic rationale for and ongoing assessment of novel investigational therapies to proficiently counsel patients regarding trial participation and the potential future role of these agents in LOPD clinical management
Course summary
Available credit: 
  • 1.00 AAPA Category I CME
  • 1.00 AMA PRA Category 1 Credit™
  • 1.00 Attendance
Course opens: 
03/29/2024
Course expires: 
03/29/2025
Cost:
$0.00
Rating: 
0
   David Kronn, MD, FACMG
   Section Chief of Medical Genetics and Director, Inherited Metabolic Disease Center
   Westchester Medical Center
   Associate Professor, Pathology and Pediatrics
   New York Medical College
   Valhalla, New York, United States
 
 
   Benedikt Schoser, MD
   Professor of Neurology
   Friedrich-Baur Institute
   Ludwig Maximilian University of Munich
   Munich, Germany
 
   Loren D.M. Peña, MD, PhD
   Professor of Pediatrics
   Cincinnati Children’s Hospital and
   University of Cincinnati School of Medicine
   Cincinnati, Ohio, United States
 
 
 

This activity is jointly provided by Medical Education Resources (MER) and Efficient LLC.

   

Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by Medical Education Resources (MER) and Efficient LLC. MER is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Designation Statement

PHYSICIAN CREDIT
Medical Education Resources designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
 
PHYSICIAN ASSISTANTS
Medical Education Resources has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for  1.00 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.
 
CERTIFICATE OF PARTICIPATION FOR OTHER HEALTHCARE PROFESSIONALS
A Certificate of Participation will be given upon completion of the activity enabling participants to register their credit with the appropriate licensing boards or associations.

Disclosure of Relevant Financial Relationships

Medical Education Resources ensures balance, independence, objectivity, and scientific rigor in all our educational activities. In accordance with this policy, MER identifies relevant financial relationships with its instructors, content managers, and other individuals who are in a position to control the content of an activity. Reported relevant financial relationships are mitigated by MER to ensure that all scientific research referred to, reported, or used in a CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. MER is committed to providing learners with high-quality CE activities that promote improvements or quality in health care and not the business interest of an ineligible company.

Planners' and Managers' Disclosure

Efficient LLC and Medical Education Resources' planners and managers have no financial relationships to disclose.

Faculty Disclosure

Dr Kronn reported the following financial relationships: 

  • Consultant or advisor for: Sanofi, AskBio
  • Speaker or member of speakers bureau for: Spark Therapeutics, Sanofi
  • Research funding from: Sanofi

Dr Schoser reported the following financial relationships: 

  • Consultant or advisor for: Amicus, Astellas, Roche Diagnostics
  • Speaker or member of speakers bureau for: Amicus, ArgenX, Astellas, Sanofi, Taysha
  • Research funding from: Alexion, Kedrion

Dr Peña reported the following financial relationships:

  • Consultant or advisor for: CVS Caremark, University of Pennsylvania
  • Research funding from: Cyclo Therapeutics, Moderna Inc., Zevra

Disclosure of Unlabeled Use/Disclaimer

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclaimer

The content and views presented in this educational activity are those of the authors and do not necessarily reflect those of Medical Education Resources, Efficient LLC, and/or Amicus Therapeutics Inc. The authors have disclosed if there is any discussion of published and/or investigational uses of agents that are not indicated by the FDA in their presentations. Before prescribing any medicine, primary references and full prescribing information should be consulted. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities. The information presented in this activity is not meant to serve as a guideline for patient management.

Method of Participation

There are no fees for participating in and receiving credit for this activity. During the period March 29, 2024 through March 29, 2025, participants must: 1) review the learning objectives and faculty disclosures; 2) complete the pre-activity assessment; 3) watch the educational activity; 4) complete the posttest by recording the best answer to each question; and 5) complete the evaluation form.

Media

Internet

Commercial Support

This continuing education activity is supported by an educational grant from Amicus Therapeutics Inc.

Available Credit

  • 1.00 AAPA Category I CME
  • 1.00 AMA PRA Category 1 Credit™
  • 1.00 Attendance

Price

Cost:
$0.00
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