Changing Tides in the Management of ASMD: Emerging Treatments and Strategies to Improve Diagnosis

Released On
March 15, 2021

Expires On
March 15, 2022

Media Type

Completion Time
60 minutes

Acute Care, Cardiology, Case Management, Education, Genetics, Hepatology, Neonatology, Neurology, Occupational Medicine, Pain Management, Pediatrics, Primary Care, Pulmonology


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

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

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)

Target Audience

This activity is intended for medical geneticists, genetic counselors, metabolic disease specialists, as well as pediatricians, family medicine providers, pulmonologists/respirologists, neurologists, hepatologists, and other specialty HCPs who may diagnose or manage ASMD/Nieman Pick.

Program Overview

Acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease, is a rare autosomal recessive lysosomal storage disorder (LSD) which can be challenging to diagnose and manage. This activity will offer access to the latest information and best practices to facilitate the early recognition of signs and symptoms and accurate diagnosis of ASMD. Furthermore, faculty will explore evolving evidence on emerging therapies and their potential role within current ASMD management approaches, as well as strategies for improved care coordination and opportunities to support patients and their families.

Learning Objectives

Upon completion of this activity, participants should be able to:

  • Identify symptoms and clinical manifestations associated with acid sphingomyelinase deficiency (ASMD), considering multiple organ systems, age of onset, and disease heterogeneity
  • Describe appropriate steps to differentially diagnose the clinical types of ASMD, including the role of biochemical testing, genetic testing, and referrals to genetics specialists
  • Explain the clinical roles of supportive care in the management of patients with ASMD
  • Evaluate the latest evidence on emerging treatments for patients with ASMD and potential clinical implications
  • Recognize resources and strategies to provide ongoing education and support to individuals with ASMD and their families



Melissa Wasserstein, MD (Course Director)

Chief, Division of Pediatric Genetic Medicine
Professor of Pediatrics and Genetics
The Children’s Hospital at Montefiore
Albert Einstein College of Medicine
Bronx, New York


Robin Lachmann, MB, BChir, PhD, FRCP

University College London Hospitals
NHS Foundation Trust
Charles Dent Metabolic Unit
National Hospital for Neurology and Neurosurgery
London, UK


Roberto Giugliani, MD, PhD

Professor, Department of Genetics
Federal University of Rio Grande do Sul
Porto Alegre, Brazil


Accreditation Statement

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.


Physician Credit Designation

AKH Inc., Advancing Knowledge in Healthcare designates this enduring 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.

Disclosures of Conflicts of Interest


Melissa Wasserstein, MD

  • Consultant: Sanofi GenzymeX
  • Contracted Research: Ara Parseghian Medical Research Fund, BioMarin, CureSanFilippo, Firely Fund, Orchard Therapeutics, Retrophin, Sanofi Genzyme, Ultragenyx

Robin Lachmann, MB, BChir, PhD, FRCP

  • Consultant and Speakers Bureau: Sanofi Genzyme

Roberto Giugliani MD, PhD

  • Consultant: Abona, Alnylam, Amicus, Biomarin, Chiesi, Denali, Inventiva, Janssen, JCR, Novartis, RegenxBio, Sanofi, Sigilon, Sobi, Takeda, Ultragenyx


Deepti Babu MS, CGC, Medical Writer: has no financial relationships to disclose.

Louis Settembrino, CHCP, Catalyst Medical Education, LLC, President and CEO: has no financial relationships to disclose.

Dorothy Caputo, MA, BSN, RN, AKH Lead Nurse Planner/Reviewer: has disclosed no financial relationships.

Patricia Brignoni, AKH Director of Operations/Reviewer: has disclosed no financial relationships.

AKH Inc. and Catalyst planners and reviewers disclosed no financial relationships

All of the relevant financial relationships listed for these individuals have been mitigated.

Instructions for Participation and Credit

There are no fees for participating and receiving CE credit for this enduring activity. To receive CE credit participants must:

  1. Read the CE information and faculty disclosures.
  2. Participate in the online activity.
  3. Submit the evaluation form.
Certificates will be emailed to the participant

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
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


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.

Disclosure of Unlabeled Use

AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education requires that faculty participating in any CE activity disclose to the audience when discussing any unlabeled or investigational use of any commercial product or device not yet approved for use in the United States.


This activity is designed for educational purposes. Participants have a responsibility to utilize this information to enhance their professional development to improve patient outcomes. Conclusions drawn by the participants should be derived from careful consideration of all available scientific information. The participant should use his/her clinical judgment, knowledge, experience, and diagnostic decision-making before applying any information, whether provided here or by others, for any professional use.

Contact Information

For CE questions please contact: