Views & Analysis Listening to ultra-rare disease communities When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges.
Views & Analysis Building our own community for an ultra-rare disease Bo Bigelow shares the story of his daughter Tess, who has an ultra-rare genetic disorder with only 51 known cases in the world.
Articles Alexion: aiming to break new ground in ultra-rare therapies Martin Mackay, head of R&D at Alexion, talks to pharmaphorum about the company's research pipeline for severe and ultra-rare diseases.
News Rezzayo antifungal okayed as Candida auris rings alarm bells Drug is first new echinocandin to be approved for marketing in the US in more than a decade.
R&D Sponsored An inside guide to unlocking clinical research in the UK An inside guide to unlocking clinical research in the UK
