It eventually led to Still being diagnosed with ATTR amyloidosis, a rare genetic condition that causes heart problems, neuropathy, and ligament and tendon troubles. Since his diagnosis in ...

The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary ...

“Launching ACT-EARLY is part of our ongoing commitment to further the genetic understanding of the variants causing ATTR and to ensure patients from around the world have access to optimal car ...

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 ...