Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments currently under review by England’s cost-effectiveness watchdog.
The FDA has marked today’s international Rare Disease Day with a number of measures to promote research and patient participation in policy and research. Among the new measures ann
Pharma company Shire and tech giant Microsoft are to team up with Eurodis, a not-for-profit group representing 700 rare disease organisations, with the goal of cutting the long wait most patients with rare conditions endure before diagnosis.
Vertex is trying reach a deal with NHS England to give patients access to all its approved cystic fibrosis (CF) drugs - and hopes it could also fund further drugs in the pipeline.
