Avidity Biosciences has filed a private placement worth around $400 million, seeking a cash infusion to help advance its pipeline of clinical-stage candidates that combine
Rare Disease Day saw the debut of a film that aims to raise awareness of cerebral adrenoleukodystrophy (cALD), a devastating and fatal condition with no pharmacological tr
Almost four years after it was approved in Europe to treat the incurable rare disease systemic amyloid light-chain (AL) amyloidosis, Johnson & Johnson’s Darzalex has b
On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.
Big pharma’s interest in using protein degradation as a therapeutic strategy shows no sign of abating, as Novo Nordisk signs a wide-ranging deal with US biotech Neomorph t
The US FDA has said it plans to deliver a decision in June on the use of Argenx’ Vyvgart Hytrulo in chronic inflammatory demyelinating polyneuropathy (CIDP), as the compan
GSK looks like it is starting to show the benefit of a revamp of its business under chief executive Emma Walmsley, with recent product launches driving a 10% rise in sales
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.