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CME / ABIM MOC

Optimizing Outcomes for Late-Onset Pompe Disease Diagnosed by Newborn Screening: An Expert Panel Summary

  • Authors: Yin-Hsiu (Nancy) Chien, MD, PhD; David Kronn, MD; Benedikt Schoser, MD, FEAN
  • CME / ABIM MOC Released: 6/14/2023
  • Valid for credit through: 6/14/2024, 11:59 PM EST
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  • Credits Available

    Physicians - maximum of 1.00 AMA PRA Category 1 Credit(s)™

    ABIM Diplomates - maximum of 1.00 ABIM MOC points

    You Are Eligible For

    • Letter of Completion
    • ABIM MOC points

Target Audience and Goal Statement

This activity is intended for cardiologists, pulmonologists, neurologists, gastroenterologists, orthopedists, and other healthcare providers who provide care to patients with Pompe disease.

The goal of this activity is for learners to be better able to determine the course of clinical management of patients with late-onset Pompe disease (LOPD) identified early through newborn screening.

Upon completion of this activity, participants will:

  • Have increased knowledge regarding the
    • Patient burden associated with LOPD after newborn screening
    • Recommended monitoring approaches for asymptomatic patients with LOPD
    • Appropriate timing for initiating enzyme replacement therapy in patients with LOPD identified through newborn screening


Disclosures

Medscape, LLC requires every individual in a position to control educational content to disclose all financial relationships with ineligible companies that have occurred within the past 24 months. Ineligible companies are organizations whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

All relevant financial relationships for anyone with the ability to control the content of this educational activity are listed below and have been mitigated. Others involved in the planning of this activity have no relevant financial relationships.

Disclosures for additional planners can be found here.


Faculty

  • Yin-Hsiu (Nancy) Chien, MD, PhD

    Clinical Professor
    Department of Medical Genetics and Pediatrics
    National Taiwan University Hospital
    Taipei, Taiwan

    Disclosures

    Yin-Hsiu (Nancy) Chien, MD, PhD, has the following relevant financial relationships:
    Consultant or advisor for: AskBio; Amicus Therapeutics, Inc.; Biogen; BioMarin Pharmaceutical, Inc.; Novartis; Sanofi
    Speaker or member of speakers bureau for: Biogen; Novartis; PTC Therapeutics; Sanofi; Takeda Pharmaceuticals North America, Inc
    Research funding from: Biogen; Sanofi

  • David Kronn, MD

    Associate Professor of Pathology and Pediatrics
    New York Medical College
    Section Chief of Medical Genetics
    Maria Fareri Children's Hospital at Westchester Medical Center
    Valhalla, New York, United States

    Disclosures

    David Kronn, MD, has no relevant financial relationships.

  • Benedikt Schoser, MD, FEAN

    Professor of Neurology 
    Friedrich-Baur Institute
    Senior Consultant
    Ludwig-Maximilians University of Munich
    Munich, Germany

    Disclosures

    Benedikt Schoser, MD, FEAN, has the following relevant financial relationships:
    Consultant or advisor for: Amicus Therapeutics, Inc.; argenX; Astellas Pharma, Inc.; Bayer; Maze Therapeutics; Sanofi
    Speaker or member of speakers bureau for: Alexion Pharmaceuticals, Inc.; Kedrion; Sanofi
    Research funding from: Amicus Therapeutics, Inc.; Astellas Pharma, Inc.

Editors

  • Maya Khalaf, MSc

    Medical Education Director, WebMD Global, LLC

    Disclosures

    Maya Khalaf, MSc, has no relevant financial relationships.

  • Megan Breuer, PhD

    Medical Writer, WebMD Global, LLC

    Disclosures

    Megan Breuer, PhD, has the following relevant financial relationships: Consultant/advisor for Paratek Pharmaceuticals, Inc. (former)

Compliance Reviewer

  • Yaisanet Oyola, MD

    Associate Director, Accreditation and Compliance, Medscape, LLC

    Disclosures

    Yaisanet Oyola, MD, has no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has no relevant financial relationships.


Accreditation Statements

Medscape

Interprofessional Continuing Education

In support of improving patient care, Medscape, LLC is jointly accredited with commendation 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.

    For Physicians

  • Medscape, LLC designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit(s)™ . Physicians should claim only the credit commensurate with the extent of their participation in the activity.

    Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit. Aggregate participant data will be shared with commercial supporters of this activity.

    The European Union of Medical Specialists (UEMS)-European Accreditation Council for Continuing Medical Education (EACCME) has an agreement of mutual recognition of continuing medical education (CME) credit with the American Medical Association (AMA). European physicians interested in converting AMA PRA Category 1 credit™ into European CME credit (ECMEC) should contact the UEMS (www.uems.eu).

    College of Family Physicians of Canada Mainpro+® participants may claim certified credits for any AMA PRA Category 1 credit(s)™, up to a maximum of 50 credits per five-year cycle. Any additional credits are eligible as non-certified credits. College of Family Physicians of Canada (CFPC) members must log into Mainpro+® to claim this activity.

    Through an agreement between the Accreditation Council for Continuing Medical Education and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

    Contact This Provider

For questions regarding the content of this activity, contact the accredited provider for this CME/CE activity noted above. For technical assistance, contact [email protected]


Instructions for Participation and Credit

There are no fees for participating in or receiving credit for this online educational activity. For information on applicability and acceptance of continuing education credit for this activity, please consult your professional licensing board.

This activity is designed to be completed within the time designated on the title page; physicians should claim only those credits that reflect the time actually spent in the activity. To successfully earn credit, participants must complete the activity online during the valid credit period that is noted on the title page. To receive AMA PRA Category 1 Credit(s)™, you must receive a minimum score of 75% on the post-test.

Follow these steps to earn CME/CE credit*:

  1. Read about the target audience, learning objectives, and author disclosures.
  2. Study the educational content online or print it out.
  3. Online, choose the best answer to each test question. To receive a certificate, you must receive a passing score as designated at the top of the test. We encourage you to complete the Activity Evaluation to provide feedback for future programming.

You may now view or print the certificate from your CME/CE Tracker. You may print the certificate, but you cannot alter it. Credits will be tallied in your CME/CE Tracker and archived for 6 years; at any point within this time period, you can print out the tally as well as the certificates from the CME/CE Tracker.

*The credit that you receive is based on your user profile.

CME / ABIM MOC

Optimizing Outcomes for Late-Onset Pompe Disease Diagnosed by Newborn Screening: An Expert Panel Summary

Authors: Yin-Hsiu (Nancy) Chien, MD, PhD; David Kronn, MD; Benedikt Schoser, MD, FEANFaculty and Disclosures

CME / ABIM MOC Released: 6/14/2023

Valid for credit through: 6/14/2024, 11:59 PM EST

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Contents of This CME Activity

To start this activity, please assess your clinical knowledge by completing the brief survey at the bottom. Answering these questions again after the activity will allow you to see what you learned and to compare your answers with those of your peers.

The Clinical Course of LOPD After Newborn Screening

Dr Schoser discusses the background of Pompe disease and the role of NBS in the early identification of individuals with LOPD.
Benedikt Schoser, MD, FEAN

An Expert Consensus on Monitoring Patients With LOPD Diagnosed Through Newborn Screening

Dr Chien illustrates the current recommendations for monitoring strategies for patients with LOPD identified through NBS.
Yin-Hsiu (Nancy) Chien, MD, PhD

An Expert Consensus on When to Start Enzyme Replacement Therapy for Patients With LOPD Identified Through Newborn Screening

Dr Kronn discusses important considerations for initiating ERT and treatment options for patients with LOPD identified through NBS.
David Kronn, MD
 

Educational Impact Challenge

The goal of this activity is for learners to be better able to determine the course of clinical management of patients with late-onset Pompe disease (LOPD) identified early through newborn screening.

Before you begin this activity, please assess your clinical knowledge by completing this brief survey. Answering these questions again after the activity will allow you to see what you learned and to compare your answers with those of your peers.

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