Learn how ATTR-CM drugs like TTR stabilizers and gene silencers work, plus their side effects and how to determine which treatment may be right for you.

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

ATTR phenotypes vary by genotype as well as by gene penetrance, age, and geographic location. A new analysis examined data from untreated, symptomatic patients who had 1 of 4 variants: F64L, I68L, ...

All forms of amyloidosis are caused by the abnormal folding of proteins in our body’s cells. In the case of wild-type ATTR amyloidosis, normal proteins accumulate but are not cleared from organs, ...

Prothena Corporation PRTA announced a collaboration with Denmark-based Novo Nordisk NVO, whereby the latter will acquire Prothena’s clinical-stage antibody, PRX004, and broader ATTR amyloidosis ...