Gene Editing Stops Nerve Damage in Rare Disease
Groundbreaking Gene Therapy Offers Hope for Hereditary Amyloidosis In a significant medical breakthrough, a single infusion of an innovative gene-editing therapy, nexiguran ziclumeran, has demonstrated remarkable and lasting reductions in the harmful protein buildup characteristic of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The findings, spearheaded by University College London’s National Amyloidosis Center in collaboration with … Read more