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
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.