Cases and Conversations: The Importance of Newborn Screening for MPS1 – How Can We Optimize Patient Outcomes From the Beginning?

Release Date: May 14, 2021
Expiration Date:  May 14, 2022

Activity Overview

Acknowledgement of Commercial Support

This activity is supported by an educational grant from Sanofi Genzyme.

 

Target Audience

This educational activity is directed toward health care professionals who regularly manage MPS I, including pediatricians, geneticists, and specialists in rare genetic disorders.

Learning Objectives

Upon successful completion of this activity, you should be better prepared to:

• Describe the pathophysiology of MPS I
• Select appropriate assessments to confirm an MPS I diagnosis based on screening results
• Analyze current and emerging therapies for the treatment of patients with MPS I
• Apply patient-centered approaches toward the management of patients with MPS I

Faculty, Staff, and Planners’ Disclosures

The staff of Physicians’ Education Resource^®, LLC, have no relevant financial relationships with ineligible entities.

Faculty

Barbara K. Burton, MD
Attending Physician
Division of Genetics, Birth Defects, and Metabolism
Ann & Robert H. Lurie Children’s Hospital of Chicago
Professor of Pediatrics
Northwestern University Feinberg School of Medicine
Chicago, IL

Disclosures:Consultant: BioMarin Pharmaceutical, Genzyme, JCR Pharmaceuticals Co, Shire Pharmaceuticals (Takeda Pharmaceutical Co); Speakers Bureau: BioMarin Pharmaceutical, Shire Pharmaceuticals.

David Kronn, MD
Associate Professor of Pediatrics
New York Medical College
Director, Inherited Metabolic Disease Center
Westchester Medical Center
Director, Biochemical Genetics Laboratory
Children and Women’s Physicians of Westchester
Chief of Medical Genetics, Inherited Metabolic and Lysosomal Storage Disorders Center
Boston Children’s Health Physicians - New York and Connecticut
Valhalla, NY

Disclosures:David Kronn, MD has no relevant financial relationships with ineligible companies.

Loren Pena, MD, PhD
Clinical Geneticist, Division of Human Genetics
Associate Professor, UC Department of Pediatrics
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH

Disclosures:Consultant: BioMarin Pharmaceutical, Sanofi (advisory board participation).

PER^® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER^® or any company that provided commercial support for this activity.