FDA OKs Burosumab for Rare Form of Rickets

APRIL 18, 2018
Cecilia Pessoa Gingerich
The US FDA this week approved burosumab (Crysvita, Ultragenyx) as the first treatment for x-linked hypophosphatemia (XLH) in adults and children ages 1 and older.

The serious condition leaves children with bowed legs, short stature, and bone and dental pain. Adults with XLH may experience joint pain, impaired mobility, tooth abscesses, and hearing loss. Adults with XLH are also at greater risk of bone fractures. In the United States, XLH affects an estimated 3000 children and 12,000 adults.

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