News FDA starts review of Regeneron's drug for rare disease FOP Can Regeneron's drug for ultra-rare bone disease FOP pick up the baton from Ipsen's Sohonos, whose growth has stalled?
News NICE backs first disease-modifying drug for ARG1 deficiency Immedica's Loargys is poised to become the first NHS therapy for ultra-rare genetic disorder ARG1 deficiency that can alter the course of the disease.
News Bankruptcy threat looms as Quince runs out of options Investor panic has poleaxed Quince Therapeutics share price, after a regulatory filing revealed the biotech's cash is running out.
News GSK departs Wave's AATD programme Wave Life Sciences has lost GSK as the partner for its alpha-1 antitrypsin deficiency drug, but other partnered RNA-editing programmes remain active.
News FDA knocks back Pharming's bid for wider Joenja use The FDA has turned down Pharming's bid to broaden the label of Joenja for rare immunodeficiency disorder APDS so it can be used in younger patients.
News Mixed data mars Sanofi's venglustat programme Sanofi has reported positive phase 3 results with venglustat in Gaucher disease, but the drug missed the mark in a Fabry disease trial.
News Remepy's hybrid Parkinson's therapy poised for phase 3 Remepy's Hybridopa therapy for Parkinson's, which combines an app with levodopa therapy, has shown efficacy in a phase 2 pilot study.
Digital Sponsored Discover a new era of healthcare panel engagement: Grounded ... A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.
