The UK lags behind other countries in getting the right treatments to rare disease patients, but promising developments are underway in a bid to help improve access
Mental health is fast becoming recognised as the missing piece of the puzzle in rare disease support
How an ongoing pricing battle between the NHS and an American biopharmaceutical giant led to a buyers’ club for cystic fibrosis drugs in the UK
Incredibly uncommon and hard to diagnose and treat, how can we advance the treatment of ultra-rare diseases if there are still so many unknowns?
Babies are tested for a number of conditions soon after birth, but varied approaches to screening worldwide highlight how fragmented the medical profession remains over this vital process
Current methods to determine the right value for orphan drugs remain controversial, yet the fast-tracking of new therapies marks a big step forward for the industry
‘If we embrace our genomic future, we stand to transform the prospects for diagnosis and treatment of rare and common diseases alike’
It is a matter of when, and not if, a pandemic will strike, so how prepared are we to combat a deadly virus infecting millions around the globe?