By Emily McKnight:

Indiana CTSI-supported research led by two Indiana University School of Medicine faculty members has culminated in U.S. Food and Drug Administration approval of a new drug for patients with an uncommon, painful and deforming bone disease.

Michael Econs and Kenneth White
IU School of Medicine researchers Michael Econs and Kenneth White (Photo courtesy of Indiana University)

The drug, burosumab, treats X-linked hypophosphatemia, or XLH. It is being brought to market by Ultragenyx Pharmaceutical Inc. in collaboration with Kyowa Hakko Kirin Co., Ltd. and its European subsidiary, Kyowa Kirin International PLC, under the brand name Crysvita.

XLH is a phosphate-wasting disease that causes rickets and osteomalacia, or softening of the bones. It is typically inherited and affects more than 12,000 patients in the United States and one out of every 20,000 people worldwide. Patients are typically diagnosed as children and have bowed legs, short stature, and experience bone pain and dental abscesses.

The Indiana CTSI provided funding and clinical research facilities to scientists and clinicians involved in this research.

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burosumab, clinical research, erik imel, IU School of Medicine, kenneth white, michael econs, new drug, rickets, translational research, x-linked hypophosphatemia, xlh