NICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family claimed the NHS cost-effectiveness body is unlawfully limiting access to a lif
BioMarin is set to file for approval for its haemophilia gene therapy valoctocogene roxaparvovec, after it achieved pre-specified clinical criteria for regulatory review.
After a year of negotiations NICE has confirmed that it is still unable to recommend Biomarin’s Batten disease drug, saying that the company is unable to address concerns about long-term ef
BioMarin Pharmaceutical has put on a stage musical to raise awareness about haemophilia, and determine whether creative expression helps patients, as it develops an experimental cure for th
