Enhancing Recognition of Late-Onset Pompe Disease to Facilitate Personalized, Multidisciplinary Care and Maintain Function: The Promise of Emerging Agents
Speakers: Loren Peña, MD, PhD, Clinical Geneticist
Duration: 60 minutes
Presented on: October 26, 2022
Loren Peña, MD, PhD
Loren Peña, MD, PhD
Clinical GeneticistLoren Peña, MD, PhD, is a clinical geneticist with over 10 years of experience in Cincinnati, Ohio and is affiliated with Cincinnati Children's Hospital Medical Center. Also, Dr. Peña is a clinical trialist who is interested in innovative approaches to treat rare disorders, particularly neurological conditions. As a clinical geneticist, she works with children who have rare and orphan diseases, lysosomal storage disorders, and metabolic disorders; also, she works in neurogenetics and gene discovery. Dr. Peña has been the principal investigator for several studies that utilize DNA and RNA-based methods to treat inherited conditions. She is glad to participate with families in the diagnostic journey and to provide hope through promising treatments for affected patients and their families.
Enhancing Recognition of Late‐Onset Pompe Disease to Facilitate Personalized, Multidisciplinary Care and Maintain Function: The Promise of Emerging Agents
ACKNOWLEDGEMENTThis activity is supported by an educational grant from Amicus Therapeutics Inc.
DATE OF RELEASE/EXPIRATIONThis activity was released on October 26, 2022 and is valid until October 26, 2023. Requests for credit must be made no later than October 26, 2023.
TARGET AUDIENCEThe educational design of this activity addresses the needs of neurologists (adult and pediatric), internal medicine physicians, primary care physicians, pediatricians, pharmacists, neurology advanced practice providers and nurses, and other clinicians who care for patients with LOPD.
STATEMENT OF NEED/PROGRAM OVERVIEWPatients with late‐onset Pompe disease (LOPD) often experience significant diagnostic delays and are susceptible to potential misdiagnoses. In addition, current treatments are associated with a number of limitations, leading to investigations of new agents that may offer greater safety, tolerability, and efficacy.
Join Dr. Loren Peña to review the symptoms of LOPD, patient factors that might influence care, and advances in the management of LOPD. Dr. Peña will discuss how to effectively incorporate new treatments, when available, into daily clinical practice and differentiate current and emerging agents to determine their appropriate place in the care of LOPD. Clinicians will be prepared to engage their patients in effective shared decision-marking to develop care plans that address the needs of their patients with LOPD.
EDUCATIONAL OBJECTIVESAfter completing this activity, the participant should be better able to:
- Recognize signs and symptoms that may raise the index of suspicion for LOPD
- Identify strategies and emerging agents to address limitations of current treatment options for LOPD
- Assess the comparative safety, efficacy, and tolerability profiles and therapeutic potential of agents being investigated for the treatment of LOPD
- Discuss the importance of engaging patients with LOPD via shared decision‐making and how it could impact outcomes
PHYSICIAN ACCREDITATION STATEMENTThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Global Education Group (Global) and PlatformQ Health Education, LLC. Global is accredited by the ACCME to provide continuing medical education for physicians.
PHYSICIAN CREDIT DESIGNATIONGlobal Education Group 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.
ABIM MOC Recognition StatementSuccessful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 medical knowledge MOC point(s) 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.
ESTIMATED TIME TO COMPLETEThis activity should take approximately 60 minutes to complete.
INSTRUCTIONS TO RECEIVE CREDITIn order to receive credit for this activity, the participant must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a score of 70% on the post-test.
HARDWARE/SOFTWARE REQUIREMENTSParticipants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.
FEE INFORMATION & REFUND/CANCELLATION POLICYThere is no fee for this educational activity.
DISCLOSURE OF CONFLICTS OF INTERESTGlobal Education Group (Global) adheres to the policies and guidelines, including the Standards for Integrity and Independence in Accredited CE, set forth to providers by the Accreditation Council for Continuing Medical Education (ACCME) and all other professional organizations, as applicable, stating those activities where continuing education credits are awarded must be balanced, independent, objective, and scientifically rigorous. All persons in a position to control the content of an accredited continuing education program provided by Global are required to disclose all financial relationships with any ineligible company within the past 24 months to Global. All financial relationships reported are identified as relevant and mitigated by Global in accordance with the Standards for Integrity and Independence in Accredited CE in advance of delivery of the activity to learners. The content of this activity was vetted by Global to assure objectivity and that the activity is free of commercial bias.
All relevant financial relationships have been mitigated.
The faculty have the following relevant financial relationships with ineligible companies:
Name of Faculty or Presenter Reported Financial Relationship Loren D.M. Peña, MD, PhD
Associate Professor of Pediatrics
Cincinnati Children’s Hospital
Nothing to disclose
The planners and managers have the following relevant financial relationships with ineligible companies:
Name of Planner or Manager Reported Financial Relationship Lindsay Borvansky Nothing to disclose Andrea Funk Nothing to disclose Liddy Knight Nothing to disclose Ashley Cann Nothing to disclose Lauren Sinclair Nothing to disclose Colleen Heffner Nothing to disclose Carole Drexel Nothing to disclose Alissa Yost Nothing to disclose
DISCLOSURE OF UNLABELED USEThis educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. Global Education Group (Global) and PlatformQ Health Education, LLC 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 any organization associated with this activity. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
DISCLAIMER:Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed in this activity should not be used by clinicians without evaluation of patient conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
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