Accreditation/Credit Designation

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

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.

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

Acknowledgment of Support

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

Cases and Conversations: The Importance of Newborn Screening for MPS1 – How Can We Optimize Patient Outcomes From the Beginning?

Release Date: May 14, 2021
Expiration Date:  May 14, 2022

Activity Overview

This online, on-demand virtual symposium brings together renowned experts in metabolic diseases to discuss mucopolysaccharidosis type I (MPS I). MPS I is a rare autosomal recessive disorder, characterized by a deficiency of α-L-iduronidase (IDUA), which results in the accumulation of the glycosaminoglycans (GAGs) dermatan and heparan sulfate in cells throughout the body. Clinical heterogeneity of MPS I is evident by the fact that symptoms can be nonspecific and may be associated with various rheumatological disorders. The irreversible nature of the symptoms and ailments caused by the accumulation and excess storage of GAGs highlights the need for timely, accurate diagnosis and early treatment intervention.

Most physicians do not have extensive knowledge or clinical experience in managing this rare disease, and their lack of experience can increase the risk of poor outcomes for patients. In this program, a panel of experts discusses the diagnosis and management of MPS 1, guided by case studies to highlight practical application.

This educational activity is an archive of the live virtual symposium held on April 15, 2021.

Acknowledgement of Commercial Support

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


Instructions for This Activity and Receiving 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.

Target Audience

This educational activity is directed toward health care professionals who regularly manage MPS I, including pediatricians, geneticists, and specialists in rare genetic disorders.

Learning Objectives

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

  • Describe the pathophysiology of MPS I
  • Select appropriate assessments to confirm an MPS I diagnosis based on screening results
  • Analyze current and emerging therapies for the treatment of patients with MPS I
  • Apply patient-centered approaches toward the management of patients with MPS I

Faculty, Staff, and Planners’ Disclosures

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


Barbara K. Burton, MD
Barbara K. Burton, MD
Attending Physician
Division of Genetics, Birth Defects, and Metabolism
Ann & Robert H. Lurie Children’s Hospital of Chicago
Professor of Pediatrics
Northwestern University Feinberg School of Medicine
Chicago, IL

Disclosures:Consultant: BioMarin Pharmaceutical, Genzyme, JCR Pharmaceuticals Co, Shire Pharmaceuticals (Takeda Pharmaceutical Co); Speakers Bureau: BioMarin Pharmaceutical, Shire Pharmaceuticals.

David Kronn, MD
David Kronn, MD
Associate Professor of Pediatrics
New York Medical College
Director, Inherited Metabolic Disease Center
Westchester Medical Center
Director, Biochemical Genetics Laboratory
Children and Women’s Physicians of Westchester
Chief of Medical Genetics, Inherited Metabolic and Lysosomal Storage Disorders Center
Boston Children’s Health Physicians - New York and Connecticut
Valhalla, NY

Disclosures:David Kronn, MD has no relevant financial relationships with ineligible companies.

Loren Pena, MD, PhD
Loren Pena, MD, PhD
Clinical Geneticist, Division of Human Genetics
Associate Professor, UC Department of Pediatrics
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH

Disclosures:Consultant: BioMarin Pharmaceutical, Sanofi (advisory board participation).

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

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.

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