The only drug that treats children with a rare degenerative disease called Batten disease could be withdrawn within the next ...

The benefits of Biomarin’s Brineura for a very rare inherited condition do not justify its price of more than £520,000 per year, NICE has said in first draft guidance. NICE had been assessing ...

The review focused on three ultra-rare disease products Kanuma, Strensiq and Brineura as well as several approved treatments for rare diseases including cystic fibrosis (CF) and pulmonary arterial ...