What Is the XLH Disease Monitoring Program?

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by Emily Malcolm |

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X-linked hypophosphatemia (XLH) is a rare genetic disease characterized by low levels of phosphate in the blood.

Because XLH is so rare, and symptoms can vary so much among patients, it’s very hard to predict what medications will be most effective for any one person, and how the disease symptoms may change over time.

The XLH Disease Monitoring Program

The XLH Disease Monitoring Program (XLH-DMP) is a study that Ultragenyx Pharmaceutical is sponsoring to monitor how XLH is affecting patients. Its results aim to help guide researchers in designing clinical trials and developing potential new treatments for XLH.

Researchers are collecting data on the 650 patients taking part in this non-interventional, 10-year clinical trial (NCT03651505). XLH-DMP is following those enrolled at multiple health centers in the U.S., Canada, and South America.

Patients in the study are tracking their symptoms and medications, as well as regularly participating in health checkups with the physicians running the study.

How will the study help physicians and patients?

In most cases, researchers publish data about XLH in small case studies, or patients share their experiences via blogs. It’s hard for physicians to look at that information and figure out what treatments will be useful. Moreover, because most small studies don’t follow patients long term, it’s hard to predict how the disease progresses.

XLH-DMP will provide more data than researchers have been able to collect thus far on XLH and its progression. Researchers are not asking patients to change their medications as part of the study, but just to record them. It well could be a rich source of information on the effectiveness of existing treatments among participants.

Are there any other studies I can participate in?

Ultragenyx is also running an online disease-monitoring program for patients with XLH and other hypophosphatemic disorders in the U.S. and Canada. The program, called BeyondXLH, is a collaboration between the company, Yale University, and the XLH Network. Like XLH-DMP, researchers ask patients to record their day-to-day symptoms and share some of their medical information.


Last updated: April 24, 2020


X-Linked Hypophosphatemia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.