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Description

The expansion of newborn screening (NBS) for Pompe disease has led to a new understanding of the greater prevalence of Pompe disease and an appreciation of earlier symptom onset in patients with late-onset variants. Symptoms can now be addressed at the earliest stages with careful monitoring, and the emergence of next-generation enzyme replacement therapy (ERT) offers clinicians the ability to prevent further disease progression. This activity will provide insights and discuss considerations around when to initiate ERT in patients diagnosed via NBS and the appropriate use of second-generation ERT.

TARGET AUDIENCE
This initiative is intended for cardiologists, genetic counselors, geneticists, neurologists, nurses, physician assistants, and primary care physicians.

AGENDA

• Welcome and Introduction
• Clinical Updates in the Treatment of LOPD: Evaluating the Latest Next-Generation ERTs
• Expanding Access to NBS: Considerations for ERT Initiation and Treatment Decision-making in LOPD
• Clinical Case Challenge: Implementing Evolving Best Practices for LOPD Diagnosed via NBS
• Q&A and Closing Remarks

LEARNING OBJECTIVES
Upon successful completion of this activity, participants should be better able to:

• Describe subsequent genetic testing and clinical assessment requirements necessary to confirm a diagnosis of late-onset Pompe disease (LOPD) in the case of positive NBS
• Assess the latest data on next-generation ERTs for LOPD, considering their use in patients who are diagnosed via NBS
• Evaluate evolving evidence and perspectives on the management and initiation of ERT in pre-symptomatic/asymptomatic patients with Pompe diseasewho have been identified via NBS
• Apply measures of disease severity/progression into clinical decisions regarding ERT initiation, selection, and optimization in LOPD diagnosed via NBS
• Develop individualized management paradigms for LOPD diagnosed via NBS, considering the latest clinical evidence

FACULTY
Priya Kishnani, MD, MBBS (Course Director)
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, NC

Ayesha Ahmad, MD
Professor of Pediatrics
Medical Director, Division of Pediatric Genetics, Metabolism and Genomic Medicine
Medical Director, Pediatric Medical Specialties Ambulatory Care Unit
Department of Pediatrics
University of Michigan
Ann Arbor, MI

Laura Case, PhD, PT, DPT, MS, PCS, C/NDT
Associate Professor in Orthopedic Surgery
Duke University School of Medicine
Durham, NC

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

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 1.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

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

This activity is supported by educational grant from Sanofi.