Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments currently under review by England’s cost-effectiveness watchdog.
The National Health Service should fund PTC Therapeutics' Duchenne Muscular Dystrophy drug, Translarna (ataluren), NICE has said, after the company agreed a discount on the list price of £2
Join the conversation, on Tuesday 28th January @ 10 am ET - 3 pm GMT - 4 pm CET, in our webinar sponsored by Syneos Health, entitled ‘Health Trends 2025: Reimagining What's Possibl