Until now, the best thing we have is an effective treatment to lessen the symptoms of the disease, but the research is still ongoing.
The gene that carries the mutation in XLH was identified in 1995, but it is not yet clear what causes this gene. The current research focuses on the processes and proteins involved in the control of phosphorus levels in the blood.
At present the laboratory Ultragenyx in collaboration with Kyowa Hakko Kirin Co., Ltd. (KHK), are working to develop KRN23, a product of research for children and adults with XLH.
The KRN23 is a monoclonal antibody fully human, discovered by KHK and administered by injection under the skin. In XLH, the underlying cause of the disease is an excess of FGF23, this antibody is designed to specifically target FGF23, join it and reduce its activity. The goal of therapy is to increase the low levels of phosphate and allow patients to make their own vitamin D. The increased levels of phosphate may improve bone health and therefore prevent or cure the rickets in children, and potentially improve osteomalacia in adults by reducing the associated symptoms of the disease.
Currently in a trial phase.
http://www.ultragenyx.com/pipeline/krn23-xlh/