New Clinical Trial Greatly Improves Genetic Rickets
A drug developed in part by Indiana University School of Medicine to alleviate the symptoms of X-linked hypophosphatemia (XLH), a disease which softens bones, has proven significantly more effective than conventional therapies.
XLH is a phosphate-wasting disease which can cause shorter stature, bowed legs and bone pain. It affects around one in every 20,000 people.
The study looked at 61 children with the condition up to 12 years old at 61 centres around the world. Each child was randomly assigned either Burosumab (an FDA-approved bi-weekly injection) or conventional therapies of oral phosphate and active vitamin D several times a day.
After 40 weeks of treatment, 72% of the children on Burosumab had seen their rickets substantially healed as compared to 6% of those on conventional therapy. The Burosumab group also saw improvements in leg deformities, distance walked in a six-minute test and active vitamin D levels.
From here, researchers intend to continue studying the long-term effects of Burosumab, including observing how the treatment affects height outcomes as an adult, and whether this treatment will reduce the necessity of surgery to correct bowed legs.