The Common and Uncommon Things - Timely Diagnosis of Mucopolysaccharidosis (MPS) I, II, and VII

Released On
December 29, 2020

Expires On
August 29, 2021

Media Type

Completion Time
30 minutes

Pediatrics, Primary Care


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Jointly provided by Postgraduate Institute for Medicine and P2P Syncro.

This activity is supported in part by independent education grants from Regenxbio, Sanofi Genzyme, and Takeda Pharmaceuticals.

Credit Available

  • Physicians — maximum of 0.50 AMA PRA Category 1 Credit(s)

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

Target Audience

This activity is intended for pediatricians in clinical practice in the US.

Statement of Need/Program Overview

What To Learn:

  • Treatment of MPS advanced, diagnosis should not be delayed.
  • Early symptoms can be common (i.e., hernia, ear infection) and uncommon (i.e., gibbus, corneal clouding, facial change)
  • Diagnostic testing can be initiated by primary care pediatricians

Learning Objectives

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

  1. Communicate that MPS I, II, and VII are lysosomal storage diseases
  2. Describe clinical features that are common to MPS I, II, and VII and with high frequency of occurrence at the time of diagnosis
  3. Explain how MPS I, II, and VII are similar or different from other types of MPS by organ and system involved
  4. Name initial diagnostic tests that can be ordered by pediatricians who are not geneticists
  5. Initiate the diagnostic process and consult geneticists when clinical suspicion is raised


Lorne Clarke, MD

Lorne Clarke, MD

Dr. Clarke is a professor of medical genetics at the University of British Columbia. He is a clinician and researcher, his team was among the original groups to define mutations at the IDUA locus in mucopolysaccharidosis type I (MPS I) patients, was the first to develop a murine model for MPS I and was actively involved in the clinical trials of enzyme replacement therapy for MPS I and actively participates in various MPS registries.

Curtis Rogers, MD

Curtis Rogers, MD

Dr. Rogers is a senior clinical geneticist and Director of the Greenville Office of the Greenwood Genetic Center in South Carolina. He received his training in both pediatrics and genetics. He has treated patients with mucopolysaccharidoses and other types of lysosomal storage disease. He has published on the genomic etiology, diagnosis and treatment of a number of genetic diseases.

Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and P2P Syncro. Postgraduate Institute for Medicine 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.

Credit Designation

The Postgraduate Institute for Medicine designates this enduring material for a maximum of 0.5 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

Postgraduate Institute for Medicine (PIM) requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy. PIM is committed to providing its learners with high quality activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest. Dr. Lorne Clarke received consulting fees from Regenxbio and Sanofi Genzyme, non-CME/CE services from Sanofi Genzyme. Dr. Rogers Curtis received non-CME/CE services from Sanofi Genzyme. The PIM planners and managers have nothing to disclose. The P2P Syncro planners and managers have nothing to disclose.

Instructions for Participation and Credit

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

  1. Read the CME/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

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient's conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer's product information, and comparison with recommendations of other authorities.

Contact Information

If you have questions regarding the certification of this activity, please contact us via email at