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 1.5 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 1.5 Contact Hours.

Acknowledgment of Support

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

Clinical Consultations™: Streamlining the Pathway From Diagnosis to Treatment in Patients With MPS 1

Release Date: December 22, 2020
Expiration Date: December 22, 2021

Activity Overview

The irreversible nature of the symptoms and ailments caused by mucopolysaccharidosis type 1 (MPS 1) increases the need for timely, accurate diagnosis and early treatment intervention. However, the clinical heterogeneity of MPS 1 means that symptoms can be nonspecific and nonfocal. This can delay the correct diagnosis, while tissue damage continues to accumulate. Diagnosis is fairly straightforward once MPS 1 is included in the differential diagnosis. Historically, treatment of MPS 1 was restricted to palliative and symptom-based interventions, but newer enzyme replacement therapy (ERT) is able to greatly slow the accumulation of mucopolysaccharides and subsequent tissue damage. Earlier diagnosis and implementation of ERT results in better outcomes for patients.

During this Clinical Consultations™, 2 expert faculty will review the symptoms and signs of MPS 1 and the diagnostic tools used to make a correct diagnosis. They will then discuss the latest evidence regarding treatment for MPS 1 and the importance of a multispecialty approach to managing patients with this disease.

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 mucopolysaccharidosis type 1 (MPS 1), including pediatricians, geneticists, and specialists in rare genetic disorders.

Learning Objectives

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

  • Outline the epidemiology, prevalence, and pathophysiology of MPS 1
  • Describe clinical presentation and initiate appropriate assessments to confirm accurate MPS 1 diagnosis
  • Review current and emerging therapies in the treatment of patients with MPS 1
  • Discuss strategies to improve treatment outcomes in patients with MPS 1

Faculty, Staff, and Planners’ Disclosures

In accordance with ACCME Guidelines, PER® has identified and resolved all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.


Barbara K. Burton, MD
Barbara K. Burton, MD
Professor of Pediatrics
Feinberg School of Medicine of Northwestern University
Attending Physician
Director, MPS/ML Treatment Center
Ann & Robert H. Lurie Children’s Hospital of Chicago
Chicago, IL

Disclosures: Grant/Research Support: BioMarin Pharmaceutical, Shire Pharmaceuticals, Alexion Pharmaceuticals, Sangamo Therapeutics, Ultragenyx Pharmaceutical, Homology Medicines; Consultant: BioMarin Pharmaceutical, Shire Pharmaceuticals, Sanofi Genzyme, Alexion Pharmaceuticals, Horizon Therapeutics, Denali Therapeutics, Aeglea Biotherapeutics, Moderna, Sio Gene Therapies, JCR Pharmaceuticals Co; Speakers’ Bureau: BioMarin Pharmaceutical, Shire Pharmaceuticals.

Shunji Tomatsu, MD, PhD
Shunji Tomatsu, MD, PhD
Principal Research Scientist
Director, Skeletal Dysplasia Research Lab
Nemours/Alfred I. duPont Hospital for Children
Wilmington, DE

Disclosures: Shunji Tomatsu, MD, PhD has no relevant financial relationships with commercial interests.

The staff of Physicians' Education Resource®, LLC (PER®) have no relevant financial relationships with commercial interests to disclose.

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 healthcare 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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