Evaluating Strategies to Improve the Recognition and Management of Mucopolysaccharoidosis Type 1

Released On
February 2, 2021

Expires On
February 2, 2022

Media Type

Completion Time
90 minutes

Genetics, Neurology, Ophthalmology, Pediatrics, Primary Care, Pulmonology, Rheumatology

Pediatrics, Respiratory

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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 independent medical education grant from Sanofi Genzyme.

Reviewed and Renewed on December 6, 2021

Credit Available

  • Physicians — maximum of 1.50 AMA PRA Category 1 Credit(s)
  • NSGC CEU — Category 2

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

Target Audience

This activity is intended for pediatricians, family medicine providers, genetic counselors, ear/nose/throat specialists (ENTs), ophthalmologists, cardiologists, orthopedists, rheumatologists, neurologists, and pulmonologists/respiratologists other healthcare providers who manage patients with MPS I.

Program Overview

This activity will offer opportunities to review symptoms and clinical manifestations of MPS I, and discuss practical approaches for screening and accurate disease diagnosis. Faculty will examine the latest evidence on current and emerging treatment options and evaluate their implications for clinical practice and patient care. Furthermore, discussions will assess strategies and best practices for implementing successful multidisciplinary management and collaborative care for patients with MPS I, including effective patient/caregiver advocacy and education.

Learning Objectives

Upon completion of this activity, the learner should be able to::

  1. Identify symptoms and clinical manifestations associated with Mucopolysaccharidosis type I (MPS I), considering multiple organ systems, time of onset, and disease heterogeneity
  2. Describe the appropriate steps to differentially diagnose attenuated and severe MPS I, including the role of newborn screening/utilization of genetic testing, and referrals to specialists and genetic counselors
  3. Explain the clinical roles of enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) and in the treatment of attenuated and severe MPS I
  4. Propose multidisciplinary, care-coordination strategies to alleviate and manage the diverse spectrum of symptoms associated with MPS I
  5. Recognize resources and strategies to provide education and support to individuals with MPSI and their families, including genetic counseling


Joseph Muenzer, MD, PhD (course director)

Professor of Pediatrics and Genetics
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina

Barbara K. Burton, MD

Professor of Pediatrics
Northwestern University
Feinberg School of Medicine
Chicago, Illinois

Timothy C. Wood, PhD

Laboratory Director
Greenwood Genetic Center
Greenwood, South Carolina

Accreditation Statement

CME Credit provided by AKH Inc., Advancing Knowledge in Healthcare

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC. AKH Inc., Advancing Knowledge in Healthcare is accredited by the ACCME to provide continuing medical education for physicians.

Credit Designation


AKH Inc., Advancing Knowledge in Healthcare designates this enduring activity for a maximum of 1.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Genetic Counselors

To apply for National Society of Genetic Counselors Category 2 CEUs, please go to https://www.nsgc.org/p/us/in

Disclosures of Conflicts of Interest


Tim Wood, PhD, FACMG has disclosed the following relevant financial relationships:

  • Consultant: Amicus
  • Contracted Research: Baebies, BioMarin, Chiesi Pharma, Sanofi/Genzyme, Ultragenyx

Barbara Burton, MD has disclosed the following relevant financial relationships:

  • Consultant: Aeglea, Alexion, Biomarin, Denali, Horizon, Inventiva, JCR Pharma, Moderna, Sanofi Genzyme, Takeda
  • Contracted Research: Denali, Homology Medicines, Sangamo, Takeda, Ultragenyx
  • Speakers Bureau: Biomarin

Joseph Muenzer, MD, PhD has disclosed the following relevant financial relationships:

  • Advisory Board: Sanofi Genzyme
  • Consultant: Sanofi Genzyme

Louis Settembrino, CHCP, President and CEO: has disclosed no relevant financial relationships.
Dorothy Caputo, MA, BSN, RN, Lead Nurse Planner/Reviewer: has disclosed no relevant financial relationships.
Patricia Brignoni, AKH Director of Operations/Reviewer: has disclosed no relevant financial relationships.

Instructions for Participation and Credit

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

  1. Read the CME/CNE information and faculty disclosures.
  2. Participate in the online activity.
  3. Receive a passing score of 70% or better on the post test.
  4. Submit the evaluation form.
Participants will be able to download the certificate.

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 include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


This course is designed solely to provide the healthcare professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the healthcare professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH, Inc., Advancing Knowledge in Healthcare specifically disclaim responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participant's misunderstanding of the content.

Disclosure Declaration

It is the policy of AKH, Inc., Advancing Knowledge in Healthcare to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The faculty must disclose to the participants any significant relationships with commercial interests 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 resolved by AKH, Inc., Advancing Knowledge in Healthcare prior to accreditation of the activity and may include any of or combination of the following: attestation to non-commercial content, notification of independent and certified CME/CE expectations, referral to National Faculty Initiative training, restriction of topic area or content, restriction to discussion of science only, amendment of content to eliminate discussion of device or technique, use of other faculty for discussion of recommendations, independent review against criteria ensuring evidence support recommendation, moderator review, and peer review. AKH, Inc., Advancing Knowledge in Healthcare planners and reviewers have no relevant financial relationships to disclose.

Contact Information

For CME/CE questions, please contact AKH at tbrignoni@akhcme.com