A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare genetic disorder. Aimee Tilley, from Kettering in Northamptonshire, was ...

Drugs firms argue that NICE ’s thresholds are too stingy. Medicines made up around 9% of health spending in Britain in 2018, ...

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for a very rare inherited condition do not justify its price of more than £520,000 per year, NICE has said in first draft guidance.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 ...

A temporary reprieve for a Vancouver Island family after their terminally-ill daughter will continue to have access to life-changing medication for two more months.

And in a separate decision, the CHMP also recommended a marketing authorisation for Biomarin’s Brineura (cerliponase alfa) for the rare neurodegenerative disorder neuronal ceroid lipofuscinosis ...

Her parents said she has seen benefits from biweekly treatments with the drug Brineura, which costs about $1 million a year. The drug is intended to slow the decline in patients’ ability to walk ...

Aimee Tilley, from Kettering in Northamptonshire, was participating in a rally in Westminster on Wednesday after being told NHS access to the drug Brineura, which slows the progress of Batten ...