There are between 6000 and 8000 rare diseases in the world, and one in ten people in Europe and the US have a rare disease; 75% of rare diseases affect children, and 30% of rare disease patients di
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