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 FDA removes barriers to cell, gene therapy development FDA wants to exert regulatory flexibility on cell and gene therapies in an effort to bring them to patients more quickly and efficiently.
Sales & Marketing Sponsored How to meet your physicians where they are online A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
