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CME: Treatment strategies in Hunter Syndrome

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.

 

Agenda

 

Introduction

Epidemiology

  • Statistics (global prevalence)
  • Mucopolysaccaridosis risk and causes
    • Pre-natal testing
    • Comorbid conditions

Pathophysiology and diagnosis [Learning objective #1 and #2]

  • Clinical features of the disease
    • Physical features
    • Respiratory complications
    • Joint stiffness
    • X-linked recessive disease pathology
    • Diagnostic methods
      • Urine GAG analysis
      • Enzyme test
      • Patient case(s)

Treatment [Learning objectives #1 and 3]

  • Prognosis of patients according to severity
  • Enzyme replacement therapy
  • Physical therapy: role in patient care
  • Patient case(s)

Potential barriers to treatment and solutions [Learning objective #4]

  • Aiding patients and their families in the patient access program

Summary, conclusions, and best practice recap

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 Shire


Release Date: June 29, 2017 -- Expiration Date: June 29, 2019

Faculty: Maria Escolar, MD

Agenda

Learning Objectives

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

  • Describe the importance of earlier treatment and diagnosis of Hunter syndrome in patients
  • Determine the likelihood of Hunter syndrome using with established diagnosis methods, given a patient case
  • Describe options available to treat 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

Accreditation

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:  Maria Escolar, MDMS, Associate Professor of Pediatrics, Director, Program for the Study of Neurodevelopment in Rare Disorders, is Pittsburgh, PA is a consultant and/or has received research grants for Alexeon, Shire, Regenxbio, Retrofin, and Aveona.

 

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

 

Commercial Support Disclosure: This program is supported by an educational grant from 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.
  • Take the post-test
  • 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.


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Fabry disease: Updates from recent research findings