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Activity Description & Educational Objectives

This educational activity based on a recent live symposium addressing the latest guidelines for treatment of phenylalanine hydroxylase deficiency (PAH), also known as phenylketonuria, and best practices for the treatment of patients across the lifespan Through a combination of case-based discussions and focused assessments of current evidence, two experts in the field offer guidance on individualized, goal-directed management of this metabolic disease.

Upon completion of this activity, participants should be better able to:

  • Describe the key challenges/unmet care needs of the different subpopulations of patients with PKU (children, adolescents, adults) and strategies to overcome them.
  • Discuss the latest clinical trial data on medical treatments for PKU, and relevant implications for practice. Establish steps to address the neuropsychological, psychosocial, developmental, growth, nutritional, and other medical needs of patients with PKU.
  • Implement best practices for optimal use of the latest dietary/nutritional interventions as well as pharmacologic and other adjuvant therapies as part of individualized, goal-directed management of PKU

Target Audience

This activity has been designed to meet the educational needs of geneticists and genetic counselors involved in the management of patients with PAH deficiency.

Requirements for Successful Completion

In order to receive credit, participants must view the activity and complete the post-test and evaluation form. There are no pre-requisites and there is no fee to participate in this activity or to receive CME credit. Statements of Credit are awarded upon successful completion of the post-test and evaluation form.

Media: Internet
Release and Expiration Dates: April 28, 2014 - April 27, 2015
Time to Complete: 90 minutes

Faculty & Disclosure / Conflict of Interest Policy

In accordance with ACCME requirements, Penn State College of Medicine has a conflict of interest policy that requires faculty to disclose relevant financial relationships related to the content of their presentations/materials. Any potential conflicts are resolved so that presentations are evidence-based and scientifically balanced.

Course Director and Moderator

Nicola Longo, MD, PhD
Professor and Chief
Medical Genetics/Pediatrics
University of Utah
Salt Lake City, Utah

Nicola Longo, MD, PhD, has a financial interest/relationship or affiliation in the form of:

Consultant for BioMarin Pharmaceutical Inc.; Hyperion Therapeutics, Inc.; and Protalix Biotherapeutics/Pfizer Inc.

Grant/Research Support from Amicus Therapeutics, Inc/GlaxoSmithKline.; BioMarin Pharmaceutical Inc.; Genzyme Corporation, a Sanofi company; Hyperion Therapeutics, Inc.; Protalix Biotherapeutics/Pfizer Inc.; and Shire.


Annette Feigenbaum, MBChB
Biochemical Geneticist
University of California, San Diego
San Diego, California

Annette Feigenbaum, MBChB, has a financial interest/relationship or affiliation in the form of:

Consultant for BioMarin Pharmaceutical Inc.

Grant/Research Support from BioMarin Pharmaceutical Inc.

Speakers Bureau participant with BioMarin Pharmaceutical Inc.

Medical Director

Christopher Washburn, PhD
PVI, PeerView Institute for Medical Education

Christopher Washburn, PhD, has no financial interests/relationships or affiliations in relation to this activity.

Other PVI staff who may potentially review content for this activity have disclosed no relevant financial relationships.

Penn State College of Medicine staff and faculty involved in the development and review of this activity have disclosed no relevant financial relationships.

Sponsorship, Credit & Support

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Penn State College of Medicine and PVI, PeerView Institute for Medical Education. Penn State College of Medicine is accredited by the ACCME to provide continuing medical education for physicians.‚Äč

The Penn State College of Medicine designates this enduring material for a maximum of 1.5 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Information about CME credit for this activity is available by contacting Penn State at 717-531-6483 or Reference course # G5462-14-T.

This CME activity is jointly sponsored by Penn State College of Medicine and PVI, PeerView Institute for Medical Education.

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

Disclosure of Unlabeled Use

The faculty of this educational activity may include discussions of products or devices that are not currently labeled for use by the FDA. Faculty members have been advised to disclose to the audience any reference to an unlabeled or investigational use.

No endorsement of unapproved products or uses is made or implied by coverage of these products or uses in our reports. No responsibility is taken for errors or omissions in reports.

Please refer to the official prescribing information for each product for discussion of approved indications, contraindications and warnings.


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