AstraZeneca has added a late-stage rare disease therapy to its pipeline, buying France’s Amolyt Pharma and its hypoparathyroidism drug eneboparatide for $800 million upfro
NICE has turned down Sanofi’s Xenpozyme as a treatment for the rare disease acid sphingomyelinase deficiency (ASMD), creating a situation where access to the drug in the U
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.
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