ultra-rare disease Archives -

When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global

Listening to ultra-rare disease communities

https://pharmaphorum.com/views-analysis-market-access/listening-to-ultra-rare-disease-communities/

Bo Bigelow shares the story of his daughter Tess, who has an ultra-rare genetic disorder with only 51

Building our own community for an ultra-rare disease

https://pharmaphorum.com/views-and-analysis/building-our-own-community-for-an-ultra-rare-disease/

Martin Mackay, head of R&D at Alexion, talks to pharmaphorum about the company’s research pipeline for severe and

Alexion: aiming to break new ground in ultra-rare therapies

https://pharmaphorum.com/articles/alexion-aiming-to-break-new-ground-in-ultra-rare-therapies/

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