Skip to main content

News

News covering rare disease

White paper on referral pathways for gene therapy

As gene therapy to treat Duchenne muscular dystrophy is approved in America, a report highlights how NHS referral for gene therapy trials, and approved treatment, need to be agreed.

Pioneering treatment highly effective for rare kidney disease

A pioneering drug for a rare kidney disease prevents organ failure and significantly improves the outcome for patients, new research has confirmed.