Listening to ultra-rare disease communitiesWhen it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global Share XListening to ultra-rare disease communitieshttps://deep-dive.pharmaphorum.com/magazine/market-access-2021/qa-ptc-therapeutics-alberto-vicent-on-listening-to-the-ultra-rare-disease-communities/
Building our own community for an ultra-rare diseaseBo Bigelow shares the story of his daughter Tess, who has an ultra-rare genetic disorder with only 51 Share XBuilding our own community for an ultra-rare diseasehttps://pharmaphorum.com/views-and-analysis/building-our-own-community-for-an-ultra-rare-disease/
Alexion: aiming to break new ground in ultra-rare therapiesMartin Mackay, head of R&D at Alexion, talks to pharmaphorum about the company’s research pipeline for severe and Share XAlexion: aiming to break new ground in ultra-rare therapieshttps://pharmaphorum.com/articles/alexion-aiming-to-break-new-ground-in-ultra-rare-therapies/
