As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.
A new pharmaceutical podcast putting health heroes in the spotlight debuts in the runup to Rare Diseases Day 2020 with an instalment focusing on Gaucher disease.
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