R&D Health Innovators – Jennifer Schranz In the rare and ultra-rare disease space, the paucity of both data and patients makes everything about research and development more complicated.
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 Akebia joins GSK in US market for oral CKD anaemia drugs The FDA has approved Akebia Therapeutics’ vadadustat as a treatment for anaemia caused by chronic kidney disease (CKD) in dialysis patients, becoming the first rival to GS
Digital Sponsored Innovation as Strategy: Enabling life sciences of the future Explore “Innovation as Strategy” framework, providing a guide for employing innovative strategies in life sciences.