Shares in Travere Therapeutics have lost more than 40% of their value after the company said that a trial designed to confirm the benefits of its recently approved Filspar
AstraZeneca has made a sizeable play in applying artificial intelligence to drug discovery in its rare disease unit Alexion, partnering with Verge Genomics in a deal that
Roche could be just months away from FDA approval of its anti-complement C5 antibody crovalimab, after the US regulator started a review of its marketing application as a
Italfarmaco's marketing application for HDAC inhibitor givinostat as a treatment for Duchenne muscular dystrophy (DMD) has been accepted by the EMA, setting up an approval
Patients with the ultra-rare disease CHAPLE now have their first FDA-approved therapy, after the regulator approved Regeneron's pozelimab for people aged one and over with
The UK's cost-effectiveness agency NICE has delivered its final verdict on Clinuvel's Scenesse, a treatment for a rare skin disorder that causes extreme sensitivity to lig
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year