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CME: Present and emerging strategies in the medical management of Hunter syndrome


Activity Description / Statement of Need:

Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), whose manifold biological roles contribute to a variety of clinical manifestations in patients presenting with MPS. MPS has seven different subcategories, of which Hunter syndrome, an X-linked recessive genetic disease, is MPS II. The syndrome is characterized by a deficiency in iduronate 2-sulfatase, which results in relatively high levels of the GAGs heparan and dermatan sulfate, leading to physical signs similar to MPS I, with the addition of aggressive behavior and developmental delay. The major complications of Hunter syndrome have to do with its widespread and varied symptoms. The nature of this disease is that multiple organ systems are impacted simultaneously, considerably decreasing patient quality of life. The most common symptoms are facial disfigurements, hepatosplenomegaly, and skeletal joint stiffness, but presentation can fluctuate widely between patients.

This activity identifies the critical components of the management process and offer solutions to close gaps in diagnosis and care, with the ultimate goals of improvement of Hunter syndrome management, treatment adherence,  and health and cost outcomes. 

Target Audience:

Pediatricians, neurologists, endocrinologists, and primary care physicians; physician assistants, nurse practitioners, and pharmacists; and any other HCPs with an interest in or who may clinically encounter patients with Hunter syndrome.

This program is supported by an educational grant from Takeda

This activity is free of charge.

Release Date: March 24, 2022 -- Expiration Date: March 24, 2024

Faculty: Natalia Gomez-Ospina, MD, PhD


Faculty introduction

Introductory content: review of Hunter syndrome

·       Epidemiology: statistics (global prevalence)

·       MPS risk and causes

o   Pre-natal testing

·       Pathophysiology, clinical presentation, disease course, and complications

·       Diagnosis and the impact of late diagnosis or misdiagnosis

o   Urine GAG analysis

o   Enzyme test

·       Patient case(s)


·       Enzyme replacement therapy, including clinical data

·       Physical therapy: role in patient care

·       Emerging treatment options

o   Gene therapy

o   Enzyme replacement therapy

·       Potential barriers to treatment and solutions, including patient access programs

·       Patient case(s)

Summary, conclusions, and best practice recap

Learning Objectives

By the end of the session the participant will be able to:

  • Describe the risks and consequences of mis- and underdiagnosis of Hunter syndrome
  • Recall strategies to improve recognition and diagnosis of Hunter syndrome
  • Discuss present approaches to treating Hunter syndrome
  • Formulate a care plan in the treatment of a patient with Hunter syndrome


ACCME Activity #202281720


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty: Natalia Gomez‐Ospina, MD, PhD, Assistant Professor, Department of Pediatrics, Division of Medical Genetics, and Division of Stem Cell and Regenerative Medicine, Stanford University, has received financial compensation from Graphite Bio and Codexis for consulting, research and speaker. She is also an employee of ImmuneAI.


Faculty WILL discuss off-label use of a commercial product.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, CEO of ScientiaCME, has no relevant financial disclosures.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Commercial Support Disclosure: This program is supported by an educational grant from Takeda.


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

Additional Courses That Are Related To This Activity

Treatment strategies in Hunter Syndrome

Child and adolescent Crohn’s disease: Updates in medical and nutritional strategies

Pediatric growth hormone deficiency (PGHD) and related disorders: Updates in recognition and treatment

Reducing vascular events and disease progression in type 2 diabetes (T2DM) and optimizing delivery of care