Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive condition that can be caused by genetic mutations or aging, and is characterized by accumulating heart damage that ultimately leads to symptoms of heart failure. In ATTR-CM,…
The most common signs of transthyretin amyloid cardiomyopathy (ATTR-CM) are those typically associated with heart failure, although problems in the nervous system or other tissues can also occur. ATTR-CM is a type of cardiac amyloidosis,…
A diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) is typically reached through a series of tests to identify signs of the disease and rule out related disorders. ATTR-CM is a type of heart amyloidosis, a group…
The treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) is a multidisciplinary effort involving interventions to slow disease progression and manage symptoms. ATTR-CM is a form of amyloidosis, where protein clumps, called amyloid deposits, accumulate in tissues.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a form of heart disease that’s typically caused by genetic mutations or aging. Determining the root cause of ATTR-CM can help guide treatment decisions, understand prognosis, and determine if family…
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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 (nex-z), in adults with hereditary transthyretin amyloidosis with polyneuropathy…
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 a new study from the University of Toronto. The findings…
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 and cardiovascular hospitalizations in people with transthyretin amyloid cardiomyopathy…
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 whether they carry disease-causing mutations. “Estimates show approximately 100,000 people…
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 transthyretin amyloidosis (ATTR-CM). Alnylam Pharmaceuticals’ Amvuttra was specifically cleared…
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 NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The…
The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage.
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, the most common cause of the disease in Taiwan, a…
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) is fully enrolled. Top-line results from part A of the…