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