A family tragedy has prompted Kay Parkinson, founder of the rare disease charity Alstrom Syndrome UK, to create a conference encouraging joined-up thinking to help improve treatments, care and serv
The FDA has rejected Pharming’s application to expand the licence of Ruconest, for a use as prophylaxis for patients with the serious rare disease hereditary angioedema (HAE).
Children in England and Wales with a rare inherited disorder causing soft and weak bones can have access to a new therapy after NICE said Kyowa Kirin’s Crysvita can receive regular NHS fund
Google Ventures and US healthcare specialist Redmile were among a group of investors supporting a £35.5 million funding round for UK biotech Evox Therapeutics, which specialises in drugs de
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.