Wednesday, November 4, 2015

Message from the Network President

From Network President Becky Mock: 

What an amazing year for the XLH Network! We’ve accomplished so much in 2015, thanks to your support. We've hired our first-ever employee, an administrative assistant who will assist with the daily operations, allowing Board members and volunteers to do more for Network members. We've established a template for XLH Days, which will make it possible to hold events in different locations around the United States.

Other critically important efforts included participation in patient advocacy and medical research conferences, where we met with numerous patient advocates and activists with the unified goal of educating, networking, and sharing best practices. We have been instrumental in assisting with recruiting patients for clinical trials this year and with distributing the Ultragenyx Burden of Illness Survey documenting the pain and suffering people with XLH endure every day, which has not been done at this level before. (It’s still open. Add your experience to the survey if you haven’t already:

We are at a critical time in the growth of the XLH Network. We must be present and visible in front of leaders of the orphan drug and disease community: National Institutes of Health, pharmaceutical industry, academia, and the FDA. Current clinical research trials for children and adults may hold the key to a promising treatment. Negotiating the lengthy FDA approval process and bringing a new treatment to patients are complicated activities and take persistence. Only at national events such as those listed above is everyone brought together to discuss the best methods of researching, diagnosing and treating rare diseases in the 56 countries in which we know our members live.

We must educate more medical professionals, especially pediatricians and dentists, about the symptoms of XLH. We need to find more ways for members to meet. And finally, we need to do more as patient advocates, raising our voices so patient centered outcome studies will be a mandatory part of all work done by researchers, clinicians, and the FDA. We must work together to make this happen.

As 2015 comes to an end, please consider making a donation to our year-end campaign:

Please help the XLH Network continue to work on behalf of everyone with XLH, their children, and their families. We can't do it without your help. Thank you!

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