Views & Analysis Duchenne's disease: A New Hope Rare diseases, exceptional people: Filippo Buccella has battled for 25 years to improve care for children with
News Contrasting fortunes for Duchenne rivals, as PTC fails and S... Two rivals in the emerging Duchenne muscular dystrophy (DMD) therapy area have experienced contrasting fortunes this week.
News Marathon halts launch of Duchenne drug after price outcry Company says it will listen to concerns of patient advocates
Views & Analysis The biggest pharma stories of 2016 so far The top 10 pharma milestones of the year to date.
News Europe delays review of PTC's Duchenne drug Review of marketing authorisation delayed, new trial likely.
News NICE backs £220,000-a-year Duchenne drug following discount 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
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends