AstraZeneca’s Voydeya has become the first oral Factor D inhibitor to be approved by the FDA as a treatment for the rare disease paroxysmal nocturnal haemoglobinuria (PNH)
AstraZeneca’s Ultomiris has been approved by the FDA to treat rare autoimmune disease neuromyelitis optica spectrum disorder (NMOSD), six months after an earlier applicati
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
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