Implementing Evidence-driven Individualized Treatment Plans within Morquio A Syndrome

This CME webcast on the management of patients with Morquio A syndrome will provide updated information and guidelines concerning the early and accurate diagnosis as well as an earlier intervention to improve patient outcomes. The activity is based on a live satellite symposium conducted during the 2015 official ACMG Annual Clinical Genetics Meeting program. Recent advances in the science of enzyme replacement therapies have presented opportunities for pharmacological interventions that improve quality of life. Clinicians will be updated on the clinical trial data and practical solutions for applying newer therapeutics to daily practice. Strategies to manage cardiopulmonary comorbidities and recommendations for the ideal clinical care model will wrap up this informative and up-to-date review of Morquio A syndrome.

Target Audience

Medical geneticists, anesthesiology, orthopedic, pulmonary, and cardiac specialists, and other healthcare professionals who manage patients within Morquio A Syndrome.

Learning Objectives

Upon completion of this activity, participants will be able to:

  1. Review the diagnostic characteristics and updated treatment guidelines for MPS IVA/Morquio A syndrome
  2. Examine pharmacologic therapeutic options for MPS IVA/MorquioA syndrome, and associated efficacy and safety data, including enzyme replacement therapeutics (ERTs)
  3. Analyze available surgical options in the treatment of MPS IVA/Morquio A, as well as methods to minimize safety risks in the case of surgical complications
  4. Develop and implement individualized treatment plans into the care of patients with MPS IVA, appropriately integrating pharmacologic and surgical options


Headshot of Doctor Clarke

Lorne A. Clarke, MD 
Professor, Medical Genetics
University of British Columbia Medical Director
Medical Director, Provincial Medical Genetics Program
Children’s and Women’s Health Center of BC
British Columbia, Canada

Headshot of Doctor Harmatz

Paul Harmatz, MD
Associate in Gastroenterology
UCSF Benioff Children’s Hospital
Oakland Campus
Oakland, California

Headshot of Doctor FongEdward W. Fong, MD
Pulmonary Medicine
UCSF Benioff Children’s Hospital
Oakland Campus
Oakland, California

Disclosure Statements

It is the policy of the Elsevier Office of Continuing Medical Education that all faculty, instructors, and planners disclose real or apparent conflicts of interest relating to the topics of this educational activity.
The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Name of Faculty or Presenter

Relationship Identified With:

Lorne A. Clarke, MD

Speaker’s Bureau: BioMarin Pharmaceutical Inc.; Genzyme Corporation; Shire Pharmaceuticals.

Paul Harmatz, MD

Consultant/Advisor: Alexion Pharmaceuticals; BioMarin Pharmaceutical Inc.; Chiesi USA, Inc; Genzyme Corporation; PTC Therapeutics; Shire Pharmaceuticals; Ultragenyx Pharmaceutical.

Grant/Research Support: BioMarin Pharmaceuticals Inc.

Edward W. Fong, MD

Consultant/Advisor: BioMarin Pharmaceuticals Inc.; Gilead Sciences, Inc.

Planners, Managers, Reviewers: Sandy Breslow; Brynne Hunter; Timothy Hayes, MD, PhD; Kristen Scollon; Emma Gilmartin; Jaimie Kelly; and Bernard Abrams, MD hereby state that they or their spouse/life partner do not have any financial relationships to products or devices with any commercials interest related to the content of this activity of any amount during the past 12 months.

Accreditation Statement

The Elsevier Office of Continuing Medical Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

Co-provided by the Elsevier Office of Continuing Medical Education and AcademicCME.

Course Open Date: May 19, 2015 | Course Expiration Date: May 18, 2016

Journal Distribution

This enduring material has been reviewed and approved for distribution by Molecular Genetics and Metabolism Journal.

Credit Designation Statement

The Elsevier Office of Continuing Medical Education designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Financial Support

This CME activity is supported by an educational grant from BioMarin Pharmaceutical Inc.

CME Inquiries/Special Needs

For all CME inquiries or special needs, please contact

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. Elsevier Office of Continuing Medical Education, AcademicCME and BioMarin Pharmaceutical Inc. do not recommend the use of any agent outside of the labeled indications.

Disclaimer: 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 on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician
Course opens: 
Course expires: 

Available Credit

  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician
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