It's not often that something life-changing happens to an entire global community, but that could be true for XLHers of all ages in 2018. Next year may see the approval, all around the world, of the first-ever treatment for XLH (and the related hypophosphatemias) that gets at the root of the problem (phosphate wasting).
But the treatment will only help if the relevant people know about it! Our next big challenge will be to reach everyone in both the patient population and the medical community and then to educate every last one of them about the realities of living with XLH, why treatment is necessary and what the treatment options are.
At the moment, there's a huge disconnect between what the experts know and what's happening in the day-to-day treatment of XLHers by non-experts. While some medical providers are current on their understanding of XLH, there are far too many instances of uninformed medical providers telling patients that there's nothing that can be done to help them (or that their symptoms are unrelated to XLH). Some patients can challenge the bad advice, but may not be able to find any better medical provider, depending on where they live. Or they may lack the knowledge or the resources to successfully challenge their medical providers' statements.
We plan to work on both sides of those conversations. Some initiatives will focus on educating the medical providers so they'll give better advice, and other projects will focus more on educating the patient community about current options so no one will be discouraged by outdated advice.
For the health care providers side of the equation, we'll be attending more medical conferences than ever, including possible going to the meetings of the American Association of Nurse Practitioners and the American Academy of Clinical Endocrinologists; and holding a Patient-Focused Drug Development meeting (attended by a representative of the FDA) in conjunction with XLH Day 2018 (in the Baltimore/Washington area) to create materials (patient testimony and guided discussions that are videotaped and then transcribed for dissemination) about the adult symptoms of XLH.
For the patient side of the equation, we'll be creating and sharing some short videos by experts on various aspects of XLH and publishing age-appropriate materials to help children understand their XLH. Plus, as we've done for the past seven years, we'll be offering the community all the resources of XLH Day, with expert speakers and a chance to network with other XLHers and their families.
But first, we need to be sure we have the resources we need to carry out these initiatives at a critical time for the XLH community. In a few weeks, we'll be participating in #GivingTuesday (November 28th), and before then we'll share the details of how you can get involved, not just by sending money, but also by sharing our Tweets and Facebook posts in your social media circles.
You don't have to wait until then, of course! We aren't waiting to start the necessary work, after all. Donations can be made here any time: https://xlhnetwork.networkforgood.com/projects/29168-every-day-giving