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 Chiesi signs $1.9bn deal to buy KalVista and its HAE drug Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.
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.
