News PTC's muscular dystrophy drug can stay on EU market - regula... But PTC must conduct further trial to confirm Translarna's benefits.
News Biogen/Ionis could launch rare disease drug within weeks Companies prepare for launch of Spinraza after phase 3 success.
News Vtesse's rare disease drug set for UK early access Vtesse drug "promising" treatment for NPC disease, says regulator.
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
