COMPLIMENTARY CME CREDIT

Tuesday, December 7th - 6:00 - 7:30 PM ET

Program Overview
This dynamic CME accredited webcast will examine the mechanism of action, using video animations, and utility of next-generation ERT and underscore strategies to facilitate timely and accurate diagnosis. Finally, this activity will utilize case-based learning to explore evolving best practices for safe and effective, individualized treatment initiation, ERT selection and switching, and ongoing management.

Pompe disease (PD) is a rare, inherited lysosomal storage disorder (LSD) resulting from aberrations in the GAA gene, leading to deficient alpha-glucosidase (GAA) activity and a toxic buildup of glycogen in muscle and other tissues. Classified into infantile-onset and late-onset (IOPD, LOPD), without enzyme replacement therapy (ERT), IOPD is fatal by age 2 and LOPD is associated with varying degrees of morbidity and mortality. Early diagnosis is critical to ensure timely ERT initiation and improve long term prognosis. Ensuring HCPs have the knowledge and skills to support prompt diagnosis and effective treatment initiation, particularly in the absence of universal newborn screening, is critical to improving outcomes, particularly in an era of advancing treatment options with improved uptake in skeletal muscles.

Target Audience
US and global inherited metabolic disease specialists, clinical geneticists, genetic counselors, pediatricians, primary care clinicians, neuromuscular specialists, cardiologists, hepatologists, pulmonologists, orthopedists, and other HCPs involved in the diagnosis and management of Pompe Disease

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

• RECOGNIZE varying clinical manifestations that may be suggestive of the different presentations of PD (ie, classical/non-classical infantile-onset [IOPD] and late-onset [LOPD]) to facilitate further diagnostic assessment
• EVALUATE the strengths, limitations, and appropriate use of available diagnostic algorithms and tools, including genetic and biochemical testing, used to make a differential diagnosis of IOPD and LOPD
• ASSESS the efficacy and safety of new and conventional ERT formulations for PD, considering their impact on cardiac, respiratory, and motor function
• APPLY strategies to efficiently diagnosis PD and initiate individualized treatment in clinical scenarios

AGENDA

• Introduction
• Enhancing Disease Recognition to Prompt Diagnosis
• Advance in Enzyme Replacement Therapy for PD
• Treating PD in Practice: Clinical Case Review
• Q&A

FACULTY
Priya S. Kishnani, MD
C.L. and Su Chen Professor of Pediatrics
Medical Director, YT and Alice Chen Pediatrics Genetics and Genomics Center|
Division Chief, Medical Genetics
Professor of Molecular Genetics and Microbiology
Duke University Medical Center
Durham, NC

David Kronn, MD, FACMG
Director, Advanced Medical Genetics, APS and Section Chief, Medical Genetics,
Westchester Medical Center
Associate Professor of Pathology and Pediatrics
New York Medical CollegeNew York, NY

Kenneth I. Berger, MD
Professor, Departments of Medicine
Neuroscience and Physiology
NYU Grossman School of Medicine
New York, NY

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 1.5 AMA PRA Category 1 Credit(s)™
• Physician Assistants — 1.5 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 1.5 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 1.5 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.

Genetic Counselor CEUs
To 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 Sanofi Genzyme.

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.

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