Cases and Conversations: Optimizing Patient-Centric Care for Chronic Acid Sphingomyelinase Deficiency: From Early Diagnosis to Emerging Treatment

Released On
May 13, 2021

Expires On
May 13, 2022

Media Type

Completion Time
90 minutes

Genetics, Pediatrics

Genetic Disorders, Metabolic Disorders

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This activity is provided by Physicians’ Education Resource®, LLC.

This activity is supported by an educational grant from Sanofi Genzyme.

Credit Available

  • Physicians — maximum of 1.5 AMA PRA Category 1 Credit(s)
  • Nurses — 1.5 Contact Hours

All other health care professionals completing this course will be issued a statement of participation.

Target Audience

This educational program is directed toward medical genetics specialists, genetic counselors, pediatricians, pulmonologists, hematologists, hepatologists, cardiologists, endocrinologists, and other clinicians involved in the diagnosis and management of ASMD.

Active Overview

Acid sphingomyelinase deficiency (ASMD) is a progressive lysosomal storage disorder associated with significant morbidity and mortality. Treatment is limited to supportive care. However, an enzyme replacement therapy, olipudase alfa, is in advanced clinical development and experts anticipate this will be the first treatment to impact underlying disease pathology. As such, education is critical to improve ASMD recognition and management. This online, on-demand virtual symposium brings together renowned experts to discuss the latest data as best practices.This educational activity is an archive of the live virtual symposium held on April 14, 2021.

Learning Objectives

Upon successful completion of this activity, you should be better prepared to:

  • Identify ASMD based on characteristic signs, symptoms, and genetic findings
  • Discuss the appropriate symptom-directed management of disease manifestations associated with ASMD
  • Describe recent data supporting the role of enzyme replacement therapy as disease-directed therapy for chronic ASMD
  • Select appropriate evidence-based management strategies for patients with ASMD based on individual goals and preferences



Margaret M. McGovern, MD, PhD

Professor and Chairman
Department of Pediatrics
Dean for Clinical Affairs
Stony Brook University School of Medicine
Stony Brook Long Island Children’s Hospital
Stony Brook, NY


Edward H. Schuchman, PhD

Genetic Disease Foundation
Francis Crick Professor of Genetics and Genomic Sciences
International Center for Types A and B Niemann-Pick Disease
Mount Sinai School of Medicine
New York, NY
UCLA Health
Los Angeles, CA


Robin Lachmann, MD, PhD

National Hospital for Neurology and Neurosurgery
University College London Hospitals
London, United Kingdom

Physician Accreditation Statement

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physician Credit Designation

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nursing Continuing Education

Physicians’ Education Resource®, LLC is approved by the California Board of Registered Nursing, Provider #16669, for 1.50 Contact Hours.

Disclosures of Conflicts of Interest

Margaret M. McGovern, MD, PhD has no relevant financial relationships with ineligible companies.

Edward H. Schuchman, PhD

  • Grant Research Support: Sanofi Genzyme; Consultant: Sanofi Genzyme
  • Speakers Bureau: Sanofi Genzyme
  • Other: Inventor on patents licensed from Mount Sinai to Sanofi Genzyme

Robin Lachmann, MD, PhD

  • Consultant: Sanofi Genzyme
  • Speakers Bureau: Sanofi Genzyme

The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible entities.

PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Instructions for Participation and Credit

  1. Complete the activity (including pre- and post-activity assessments).
  2. Answer the evaluation questions.
  3. Request credit using the drop-down menu.
You may immediately download your certificate.

Course Viewing Requirements

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Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.