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Optimizing Outcomes in Late-Onset Pompe Disease: Integrating New Therapies, Whole-Person Markers for Disease Monitoring, and Shared Decision-making Into Practice

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Description



Please Register Below for this FREE Accredited-CE Activity Webinar -

Thursday March 14, 2024 - 5:00 - 6:00 PM EST

Program Overview
With the approval of 2 new entities, recent advances in enzyme replacement therapy (ERT) have created a paradigm shift in which healthcare providers (HCPs) have gone from only being able to use a single agent to being able to choose a treatment from multiple available options. Furthermore, the increasing adoption of newborn screening is allowing for greater appreciation of the early manifestations of late-onset Pompe disease (LOPD) and the opportunity to intervene earlier in the disease course. This activity will explore the new understanding around LOPD symptom onset and the efficacy of next-generation treatments to modify disease progression. 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.


Target Audience
This activity is intended for neuromuscular specialists, neurologists, clinical geneticists, genetic counselors, inherited metabolic disease/lysosomal storage disorder specialists, as well as other HCPs involved in the diagnosis and management of LOPD.

Educational Objectives
Upon completion of this activity, the learner should be able to:

  • Recognize the clinical impact that the progressive, multisystemic pathology of LOPD has on treatment initiation and effectiveness
  • Evaluate clinical data, considering varying efficacy and safety outcomes, on ERT/enzyme stabilizer therapy and second generation ERT in LOPD
  • Integrate varying measures of disease severity and progression, including patient-reported outcomes, into individualized treatment decision-making for LOPD
  • Identify strategies to support shared decision-making as part of treatment selection and management decisions in LOPD
  • Develop individualized LOPD treatment plans that include next-generation therapies and consider the latest clinical data, holistic measures of disease progression, and patient preferences
AGENDA
  • The Impact of Our Evolving Understanding of LOPD on Treatment Goals
  • Evaluating the Utility of Next-generation LOPD-Modifying Therapy
  • Delivering Comprehensive Care for LOPD in an Evolving Treatment Era
  • Q&A and Closing Remarks
FACULTY
Priya S. Kishnani, MD, MBBS
C.L. and Su Chen Professor of Pediatrics

Medical Director, YT and Alice Chen
Pediatric Genetics and Genomics Center
Division Chief, Medical Genetics
Professor of Molecular Genetics and Microbiology
Duke University Medical Center
Durham, North Carolina

Professor Mark Roberts, BSc, MBChB, FRCP, MD
Consultant Neurologist
Greater Manchester Neurosciences Unit
Salford Royal NHS Foundation Trust
Salford, United Kingdom

Professor Dr. med. Benedikt Schoser, FEAN
Friedrich Baur Institute
Neurological Clinic and Polyclinic
LMU Clinic
Ludwig-Maximilians-University Munich
Munich, Germany

Disclosure
It is the policy of AKH Inc. to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The author must disclose to the participants any significant relationships with ineligible companies whose products or devices may be mentioned in the activity or with the commercial supporter of this continuing education activity. Identified conflicts of interest are mitigated by AKH prior to accreditation of the activity. AKH planners and reviewers have no relevant financial relationships to disclose.

CRITERIA FOR SUCCESS
Certificates of completion will be awarded based on the participant’s attendance and submission of the activity evaluation/claim credit form. You must participate in the entire activity to receive credit. There is no fee to participate in this activity. If you have questions about this activity, please contact AKH Inc. at tbrignoni@akhcme.com.

Credit provided by AKH Inc., Advancing Knowledge in Healthcare
Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Physician Assistants — 1.0 AAPA Category 1 CME credits
  • NSGC CEU — Category 2
All other health care professionals completing this course will be issued a statement of participation.

In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC. AKH Inc., Advancing Knowledge in Healthcare 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.

Physicians

AKH Inc., Advancing Knowledge in Healthcare designates this live activity 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.

Physician Assistants
AKH Inc., Advancing Knowledge in Healthcare 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 1.0 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.


Genetic Counselor

CEUsTo apply for National Society of Genetic Counselors Category 2 CEU, please go to https://www.nsgc.org/Education-and-Events/Continuing-Education-Unit-CEU-Information

Provider Information

This program is jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC

Commercial Support:This activity is supported by an educational grant from Amicus Therapeutics, Inc.

Disclaimer
This course is designed solely to provide the healthcare professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the healthcare professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH Inc. specifically disclaim responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participant's misunderstanding of the content.

Disclosure of Unlabeled Use and Investigational Product

This educational activity may include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

AKH Privacy Policy (link:
https://akhcme.com/akhcme/pages/privacy)

March 14, 2024
Thu 5:00 PM EDT

Duration 1H 0M

This live web event has ended.

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