Clinical Briefings™: Diagnosing and Treating MPS I: Opportunities to Improve Prognosis

Released On
November 27, 2019

Expires On
November 27, 2020

Media Type

Completion Time
90 minutes

Cardiology, Genetics, Neonatology, Ophthalmology, Otolaryngology, Pediatrics, Primary Care, Pulmonology


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

Supported by an educational grant from Sanofi Genzyme.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Nurses — maximum of 1.0 contact hours.

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

Target Audience

This online educational activity is directed toward pediatricians and medical specialists who have the opportunity to recognize MPS 1 such as ophthalmologists, growth specialists, orthopedic surgeons, general surgeons, ear nose and throat specialists, cardiologists, pulmonologists, geneticists. Additionally, nurse practitioners (NPs), physician assistants (PAs), nurses and other health care professionals who would be involved in the diagnosis and management of MPS I will be invited to participate.

Program Overview

This CME online activity, Clinical Briefings™: Diagnosing and Treating MPS I: Opportunities to Improve Prognosis, features written content complimented by audio commentary from Paul Orchard, MD, a pediatrician and expert in the field of cell therapies for inherited metabolic diseases. This activity provides an overview of lysosomal storage disorders with a focus on mucopolysaccharidosis type I (MPS I), characteristic features and diagnostic assessment in this setting, and current and novel treatments for individuals who have received a diagnosis of this disease.

Benefits of Participating

  • Recognize characteristic features of MPS I for early diagnosis
  • Determine newborn screening recommendations in the state where you practice
  • Explain care decisions to patients and caregivers
  • Discuss clinical data of new therapies for MPS I with your colleagues
  • Learn something new about lysosomal storage disorders and earn continuing medical education (CME) credit.

Learning Objectives

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

  • Review the pathophysiology of MPS I
  • Recognize the clinical presentation and diagnostic evaluation of MPS I
  • Evaluate the limitations in the historical standard of care
  • Discuss the efficacy and safety of enzyme replacement therapy and emerging therapies for patients with MPS I


Paul Orchard, MD

Medical Director of the Inherited Metabolic & Storage Disease Bone Marrow Transplantation Program Professor
Division of Pediatric Bone Marrow Transplantation
University of Minnesota
Minneapolis, MN

Accreditation Statement

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

Credit Designation

Physicians’ Education Resource®, LLC, designates this enduring material 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.

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.0 Contact Hour.

Disclosures of Conflicts of Interest

Paul Orchard, MD

  • Grant Research Support: Magenta Therapeutics, Immusoft Corporation, bluebird bio.

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

Method of Participation

Instructions for This Activity and Receiving Credit

  1. You will need to log in to participate in the activity.
  2. Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review audio files/content until you finish the presentation.
  3. At the end of the activity, educational content/audio files will be available for your reference.
  4. In order to receive a CME/CE certificate, you must complete the activity.
  5. Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” You may immediately download a CME/CE certificate upon completion of these steps.

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.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, it is the policy of PER® to ensure fair balance, independence, objectivity, and scientific rigor in all of its CME/CE activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME/CE activity to disclose all relevant financial relationships with commercial interests. The ACCME defines “relevant financial relationships” as financial relationships in any amount occurring within the past 12 months that creates a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.

Disclosure of Unlabeled Use

This CME/CE activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME/CE activity is for continuing medical and nursing education purposes only and is not meant to substitute for the independent clinical judgment of a physician and nurse relative to diagnostic or treatment 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 the company that provided commercial support.