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
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.