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 NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
