News BioMarin halts Voxzogo trials due to safety signal BioMarin has suspended dosing in phase 2 trials of its achondroplasia therapy Voxzogo aimed at broadening the drug's label.
News FDA approves leucovorin – but not for autism The FDA stops short of approving leucovorin for autism, but will its clearance for ultra-rare condition CFD-FOLR1 lead to more off-label prescribing?
News Alfasigma licenses liver drug from GSK in $690m deal With an FDA verdict looming, GSK has signed an eleventh-hour deal to give Alfasigma rights to its primary biliary cholangitis therapy linerixibat.
News PepGen slumps as FDA places hold on lead drug Shares in PepGen fell sharply after the FDA placed a partial clinical hold on its lead drug for rare neuromuscular disease DM1.
News Ascendis scores FDA approval for weekly dwarfism drug Children with achondroplasia will soon have the option of a once-weekly - rather than daily - injection, courtesy of Ascendis' newly approved Yuviwel.
News New biotech Atrium born out of Novartis/Avidity marriage Atrium Therapeutics has launched with $270m in funding and two preclinical siRNA candidates for rare genetic cardiomyopathies.
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.
