Clinical Briefings™: X-Linked Hypophosphatemia: Diagnosing and Addressing the Underlying Cause of the Disease

Released On
June 20, 2019

Expires On
June 30, 2020

Media Type

Completion Time
60 minutes

Genetics, Rare Disease


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This activity is provided by Physicians' Education Resource®, LLC.

Supported by an educational grant from Ultragenyx Pharmaceutical Inc.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)

All other health care professionals completing this course will be issued a statement of participation.

Target Audience

This online educational activity is directed toward primary care physicians, pediatricians, endocrinologists, nephrologists, dentists, nurse practitioners (NPs), physician assistants (PAs), nurses and other healthcare professionals who would be involved in the diagnosis and management of XLH.

Program Overview

X-linked hypophosphatemia (XLH) is a lifelong condition that requires long-term treatment and monitoring. Clinical presentation is variable, causing significant morbidity in patients with XLH as they age. This morbidity may result from impairment in mobility, osteoarthritis, enthesopathy, stress fractures in weakened bones, and pain and discomfort. Current treatment consists of conventional therapy, which includes vitamin D supplementation and oral phosphate, aimed at improving growth, lessening the severity of symptoms, and preventing skeletal abnormalities; and burosumab, an anti-FGF23 fully human monoclonal antibody, and the first-in-class treatment targeting the underlying pathophysiology of XLH.

This activity has recently been updated to include the latest data from the 64-week extension trial, which found that burosumab exhibited superior improvement of bone disease in children with XLH as compared to conventional therapy using oral phosphate and active vitamin D. As a result of the addition of new data, Physicians' Education Resource®, LLC, has extended the accreditation for this program from December 31, 2019, to June 30, 2020.

Learning Objectives

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

  • Utilize clinical trial data concerning novel therapy for X-linked hypophosphatemia (XLH) in clinical care of patients
  • Evaluate the medical needs of patients with XLH and appropriate multidisciplinary approach to care
  • Apply the knowledge of adverse drug reactions, toxicities, and relative risks of pharmacologic and nonpharmacologic therapies to select appropriate interventions


Thomas O. Carpenter, MD

TProfessor of Pediatrics
Professor of Orthopedics and Rehabilitation
Yale University School of Medicine
New Haven, Connecticut

Accreditation Statement

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians and is a provider approved by the California Board of Registered Nursing (CBRN).

Credit Designation Statement

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit(s)™ and for 1.0 contact hours for nurses. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosures of Conflicts of Interest

The following contributors have relevant financial relationships with commercial interests to disclose:

Thomas O. Carpenter, MD

  • Grant/Research Support: Ultragenyx Pharmaceutical (Investigator in clinical trials)
  • Consultant: Clementia, Inozyme, Pharmacosmos, Ultragenyx Pharmaceutical
  • Spouse’s employment role: Consultant for Thermo-Fisher
  • Investigational/Off-Label Use of Drugs/Devices Disclosure: Alfacalcidol; Calcitriol; various oral phosphate salts

Method of Participation

There are no fees to participate in the activity. Participants must review all activity information, including the learning objectives, disclosure statements, and content. To receive credit for participation, participants must complete the post-test (achieving a passing grade) and program evaluation. Certificates can be printed immediately.

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above.

Disclaimer/Disclosure of Unlabeled Use

This activity may or may not discuss investigational, unapproved, or off-label uses of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME activity is for continuing medical education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to the diagnostic, treatment, and 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 Physicians’ Education Resource®, LLC, or any of the companies that provided commercial support for this activity.