NHS "isn't delivering equitable care for rare diseases"
A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
Newsletters and Deep Dive digital magazine
A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.
Eisai has launched a 'digital destination', called Kompass, that aims to support patients after they have been given a diagnosis of kidney cancer.
NHS patients in England with vitiligo will soon be able to access Incyte's Opzelura, the first drug shown to reverse the skin-lightening disorder.
The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.
Editor's Picks
Newsletters and Deep Dive
digital magazine