Infants with a rare and fatal congenital disorder called Wolman disease in England and Wales will soon have access to a new drug via the NHS that can dramatically extend t
AstraZeneca has made a sizeable play in applying artificial intelligence to drug discovery in its rare disease unit Alexion, partnering with Verge Genomics in a deal that
AstraZeneca's Soliris has become the first approved treatment in the EU for children and adolescents with generalised myasthenia gravis, a disorder that causes weakness in
Novartis has a second positive phase 3 trial in the bag for its targeted factor B inhibitor iptacopan, vying to become an oral alternative to injectable therapies for ultra-rare blood disor
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
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year
