HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

FDA Approves XLH Treatment with Crysvita for Patients 6 Months and Older

The U.S. Food and Drug Administration (FDA) approved a label update for Crysvita (burosumab) to cover treatment of X-linked hypophosphatemia (XLH) for patients 6 months and older. This update includes data showing improvement in stiffness, continued healing of fractures, and maintenance of efficacy with longer-term use of Crysvita…