The XLH Network, Inc. has been awarded a grant to establish a Registry and Natural History Study in collaboration with the National Organization for Rare Disorders and the U.S. Food and Drug Administration. You can read the official NORD press release here: http://rarediseases.org/nord-announces-20-rare-disease-patient-groups-selected-to-develop-natural-history-studies-as-part-of-fda-cooperative-agreement/
The importance of this type of study cannot be emphasized enough. The "natural history" of a disorder is its progression, from inception to resolution. XLH, like many rare disorders, has never been the subject of a rigorous natural history study, so there is a great deal of uncertainty, particularly about the symptoms in adults, but also with many aspects of childhood treatment. The lack of scientific data makes it difficult for both patients and doctors to make informed decisions about treatment.
This grant will provide us with a customizable, expandable framework to begin gathering the data that researchers and health care providers can use to improve the treatment of XLH patients world-wide.
This is a long-term project and a demanding one, in terms of both human and financial resources. The Network has begun gathering these resources to undertake the work, thanks to the leadership of my predecessor and the many contributions of the board of directors, the scientific advisory board, and our other generous volunteers and donors.
Still, there is a great deal of work to be done and money to be raised to pay for this new and game-changing project. We could use your support now, more than ever. If you'd like to make a donation, the information you'll need is here: http://xlhnetwork.org/index.php/donate/.