ATTR-CM

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a Phase 3 clinical trial testing the investigational gene-editing therapy, nexiguran ziclumeran…

More than half of adults with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), a condition marked by heart damage, also have multi-nerve damage, according to…

Treatments that target the transthyretin (TTR) protein, either by stabilizing it or reducing its production, were associated with about a one-third lower risk of death…

The European Commission has expanded the indication of Amvuttra (vutrisiran) to include adults with ATTR amyloidosis with cardiomyopathy, or heart damage (ATTR-CM), regardless of…

The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary…

Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy…

The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment…

Treatment with Vyndaqel (tafamidis) reduces signs of heart damage for most people with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) associated with the A97S mutation,…

Eidos Therapeutics announced that the Phase 3 clinical trial evaluating its lead candidate acoramidis (formerly known as AG10) in treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM)…