Flagship Journal

Rare diseases are increasingly recognised as a global public health priority.1 Whilst rare diseases have a low prevalence individually, it is estimated…

Rheumatoid arthritis (RA) is a systemic autoimmune condition prevalent in North America and Northern European populations with an incidence of 1%…

As the mediator of gas exchange, the lung is an essential organ for mammalian survival and has thus evolved a diverse and intricate set of defence…

Interstitial cystitis/bladder pain syndrome (IC/BPS), is among one of the most common diagnoses that give rise to chronic pelvic pain…

For many people with Crohn’s disease (CD), onset occurs in childhood or adolescence. Treatment for CD has moved from predominantly surgical to…

WHAT is the value of newborn screening (NBS) to the rare diseases community?…

ARTIFICIAL intelligence could one day form the backbone of healthcare, but with such a powerful technology looming on our horizon, what exactly does…

New trends in clinical research and trial design were discussed, as well as challenges associated with the need to perform innovatively to advance…