We'd like to suggest that you think about some people you probably haven't met: the researchers working on a cure for XLH. They need our help.
Until recently, research was done without much consideration of the patients' day-to-day needs. While a great deal of good work was done, it's not the only way to proceed and may well not be the most efficient way to proceed. Finally, patients are being given the opportunity to contribute to the research in a variety of ways.
At the moment, we in the XLH community have three opportunities to participate in such patient-centered research.
The newest one is the Rudy study, now open for registration to all adults and children with XLH who live in the UK. The aim of the study is to improve understanding of all aspects of rare bone diseases with the aim of developing new tests and treatments to improve patients’ lives. Rudy is led by a research team at the University of Oxford which is funded by the NIHR Rare diseases of Bone, Joint and blood and the Oxford NIHR Musculoskeletal Biomedical Research Unit, University of Oxford. If you are interested in finding out more including how to register please check the library on their website www.rudystudy.org.
The second opportunity i s coming to a close just as the Rudy study begins. It's the Burden of Illness study that is available to patients, both adults and children, worldwide. You can read more about the study here: https://www.amihealthy.com/custom/ultragenyx/burdenofillness/burdenofillnesshome.aspx?SITE=XLHNETWORK
Please note that this is your last chance to participate in the Burden of Illness study. December 8, 2014 is the last date for the study to accept information.
Another current opportunity is the "graduates" study by Dr. Whyte, which is open to patients who were treated as children at the Shriner's Hospital in St. Louis. If you're eligible for that study, you can read about it here: http://xlhnetwork.org/files/9613/9931/2887/Graduate_Study_Flyer_Edited2_1.pdf
As always, the XLH Network, Inc. does not counsel individual patients either for or against participation in any specific research study. Prospective volunteers should always carefully review the study's documentation, and discuss the pros and cons of their participation with trusted advisors, including their health care providers and family.