University College London’s National Amyloidosis Center leads a multinational team reporting that a single infusion of an in vivo gene-editing therapy (nexiguran ziclumeran) produced rapid, deep, and durable reductions in serum transthyretin for hereditary transthyretin amyloidosis with polyneuropathy, with disease measures largely stable or improved through 24 months.
This article was originally published on MedicalXpress.com