There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition, according to the N
Santhera has completed a rolling application for its Duchenne muscular dystrophy (DMD) therapy vamorolone in the US, setting up a possible approval and launch in the latter half of 2023.
Less than a week before Ipsen's palovarotene was due to go in front of an FDA advisory committee, the meeting has been called off following a request by the regulator for more data.
Novartis has chalked up another clinical trial win for its targeted factor B inhibitor iptacopan as it strives to position the drug as a patient-friendly, oral alternative to big-selling in
Patients across the UK with chronic immune thrombocytopenia (ITP) now have another treatment option, after NICE backed use of Spanish drugmaker Grifols' Tavlesse for adults with the rare bl