News FDA clears Neurocrine drug for rare adrenal disorder Neurocrine Bioscience's Crenessity is the first new option for classic congenital adrenal hyperplasia (CAH) in more than 70 years.
News Novo sets aside $1.2bn for rare diseases plant Novo Nordisk has started building its first brand-new manufacturing facility in Denmark this century and will spend around $1.2bn on the plant.
News Refiling for cALD therapy on the cards after new data Readout from NEXUS study sets up mid-2025 refiling for Minoryx and Neuraxpharm's leriglitazone for rare disease cerebral adrenoleukodystrophy (cALD).
News Warning letter adds to Applied Tx run of bad news Applied Therapeutics gets a warning letter from the FDA over a trial of rare disease drug govorestat, which was rejected by the agency last week.
News Applied Tx craters as FDA rejects lead product candidate Applied Therapeutics plummets after the FDA turns down its marketing application for govorestat as a treatment for galactosaemia
News FDA probing cancer cases with Bluebird's CALD gene therapy The FDA is scrutinising reports of haematological cancers in patients treated with bluebird bio's Skysona gene therapy for neurological disease CALD
Spotlight on World Without Disease initiative: 2024 update 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
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