Alexion is preparing filings in major markets for its rare disease drug Soliris in a new indication after a positive readout in a rare neurological disease.
Spark Therapeutics’ and Novartis Luxturna gene therapy for a rare inherited eye disease has been okayed by European regulators, paving the way for an EU licence in the coming months.
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