www.fdanews.com/articles/188838-nhs-makes-crysvita-available-for-treatment-of-rare-genetic-disorder
NHS Makes Crysvita Available for Treatment of Rare Genetic Disorder
October 17, 2018
The UK’s National Health Service announced that Kyowa Kirin’s Crysvita will be available as a treatment for the inherited genetic disorder X-linked hypophosphatemia (XLH) in children whose bones are still growing.
The NHS issued final guidance based on a recommendation by the National Institute for Health and Care Excellence. NICE initially recommended against funding for Crysvita, but changed its mind in September.
XLH causes low phosphate levels in the blood, leading to weak, fragile bones that can cause disabilities and pain. Children with the disorder may also suffer hearing loss and dental problems.