Diagnosis is just the beginning of the battle

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

How rare diseases can impact mental health

Mental health is fast becoming recognised as the missing piece of the puzzle in rare disease support

Why buyers’ clubs are the last option for desperate families

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

Ultra-rare disease diagnosis is an uphill battle

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?

How better genetic screening could save babies’ lives

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

Why are orphan drugs so expensive?

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

It’s time to make rare diseases a priority

‘If we embrace our genomic future, we stand to transform the prospects for diagnosis and treatment of rare and common diseases alike’

Preparing for the next pandemic

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?