An initiative set up by the FDA to accelerate the development of therapies for rare disease in the same way that Operation Warp Speed delivered COVID-19 vaccine has chosen
Rare diseases are complex, but the need is urgent. Alexion’s Eunice Alvazzi discusses the shared responsibility in achieving access to innovation in rare diseases
While at Reuters Pharma Europe in Barcelona the other week, pharmaphorum web editor Nicole Raleigh spoke with Florian Schnappauf, VP of enterprise commercial strategy at Veeva Europe, about
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
To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.
Gilead Sciences has beefed up its pipeline with an agreement to buy CymaBay Therapeutics and its seladelpar drug for rare liver disease primary biliary cholangitis (PBC),
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