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.
Oncology Treating primary bone cancer: The potential of listeria-base... In a new pharmaphorum podcast, Paul Romness, president and CEO of OS Therapies, discusses the company's work in osteosarcoma.
Market Access The value of a partner, globally and locally A new podcast looks at recent trends related to drug approvals, as well as challenges and opportunities both small and large pharma companies face
