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
Due to the nature of their conditions, people living with rare diseases experience a wide, endless range of emotions and feelings, including sadness, grief, shame, loneliness, jealousy, ang
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
Bavarian Nordic is preparing to file for regulatory approval of a vaccine against chikungunya virus after its CHIKV VLP candidate showed protective efficacy in a second ph
To improve trial recruitment, engagement, and retention, and to increase the likelihood that trials reflect what matters most to patients, regulatory authorities increasingly point to metho