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
European regulators have granted a fast path to market for Calliditas Therapeutics’ potential rare kidney disease drug, Nefecon, weeks after a similar decision by the FDA.
Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.