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CME: Treatment strategies in Hunter Syndrome

ACCREDITATION EXPIRED: October 02, 2022

Activity Description / Statement of Need:

In this online CME self-learning program:

Mucopolysaccaridoses (MPS) are group of genetic disease characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), whose manifold biological roles resulting in a variety of clinical manifestations in patients presenting with MPS. MPS has seven different subcategories, of which Hunter syndrome is MPS II. A deficiency in iduronate 2-sulfatase results in relatively high levels of the GAGs heparan and dermatan sulfate, resulting in physical signs similar to MPS I with the addition of aggressive behavior and developmental delay. Hunter syndrome is an X-linked recessive genetic disease, with males more likely to develop disease and females more likely to be carriers.

Target Audience:

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


This program is supported by an educational grant from Takeda/Shire.

Learners may participate in this activity free of charge.


Release Date: October 02, 2020 -- Expiration Date: October 02, 2022

Faculty: Natalia Gomez-Ospina, MD, PhD

Agenda

Introduction

Introduction content: cursory refresher and review of Hunter syndrome

· Epidemiology: Statistics (global prevalence)

· MPS risk and causes

    • Pre-natal testing

· Pathophysiology, clinical presentation, and disease course

· Comorbid conditions

· Diagnostic methods

    • Urine GAG analysis
    • Enzyme test

· Patient case(s)

    • Treatment

· Enzyme replacement therapy, including clinical data

· Physical Therapy: Role in patient care

· Emerging treatment options

  • Gene therapy
  • 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:

  • Determine the likelihood of a Hunter syndrome diagnosis using established diagnostic methods, given a patient case.
  • Describe current and investigative options available for management of Hunter syndrome, and design a medical plan to treat a patient with Hunter syndrome.
  • Describe present barriers to care in the treatment of patients with Hunter syndrome.
  • Describe the clinical outcomes associated with Hunter syndrome treatment.

Accreditation

ACCME Activity #201540546

ACCREDITATION FOR THIS COURSE HAS EXPIRED. YOU MAY VIEW THE PROGRAM, BUT CME / CE IS NO LONGER AVAILABLE AND NO CERTIFICATE WILL BE ISSUED.

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 Disclosure: Natalia Gomez-Ospina, MD, PhD, Assistant Professor of Pediatrics, Stanford University, has recieved funding as a consultant for Codexis, Inc. and is on the scientific advisory board for Integral Medicines.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, CEO of ScientiaCME, has no relevant conflicts of interest to disclose.

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

Instructions

  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowedge 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 and Linguistic Competence

System Requirements

PC
Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
MAC
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

Lysosomal Storage Disorders: Updates from recent research findings

Advances in the management of Fabry disease

Gaucher disease: Updates from recent research findings

Fabry disease: Updates from recent research findings