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
It’s a case of so far, so good for Ipsen with its recent acquisition of Albireo, after the main asset in the deal – Bylvay – secured FDA approval for its second rare disea
Astellas’ gene therapy for rare disease X-linked myotubular myopathy (XLMTM) has been hampered by safety concerns, so its decision to license an alternative from start-up
Join the conversation, on Tuesday 28th January @ 10 am ET - 3 pm GMT - 4 pm CET, in our webinar sponsored by Syneos Health, entitled ‘Health Trends 2025: Reimagining What's Possibl