News FDA maps new regulatory route for ultra-rare diseases The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.
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.
Patients LSX2026: On promise and patience, with Hans Schambye At LSX World Congress 2026 in Lisbon, Portugal, web editor Nicole Raleigh spoke with Hans Schambye, CEO of BOOST Pharma.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
