Global Blood Therapeutics has seen its sickle cell disease drug approved by the FDA, promising to be the first treatment targeting the root cause of the condition.
GP Karishma Patel tells us about the daily challenges of living with the ultra-rare condition Familial Chylomicronaemia Syndrome (FCS), which prevents the body from breaking down fats.
Ipsen has added to its rare diseases pipeline after striking an agreement with US biotech Blueprint for a licence to develop and market a drug for the rare disease fibrodysplasia ossifican
UCB is to buy US biotech Ra Pharma in a deal worth around $2.1 billion, adding a late-stage treatment for the rare muscle weakness disease myasthenia gravis to its R&D pipeline and sett
The UK health secretary has responded to an open letter from rare disease patients, raising concerns about the country’s involvement in EU health research networks after Brexit – but his re
