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.

Rare disease groups: scaling up from the kitchen table

People wanting to establish rare disease support groups face lack of understanding and awareness from the medical profession, challenges finding others with the same condition, often little fun

Funding rare disease treatments in the UK – navigating the l...

With the appraisal process for highly specialised treatments in the UK described by one NHS insider as 'impenetrable', and The MPS Society's (The Society for Mucopolysaccharide Diseases) recent

Available on-demand: The Price is Right? - Delivering UK acc...