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
Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.