Hereditary transthyretin-mediated (hATTR) amyloidosis is an inherited and progressively debilitating disease caused by mutations in the TTR gene.

Mutations in the TTR gene cause abnormal amyloid proteins to accumulate, causing peripheral neuropathy, autonomic neuropathy, and cardiomyopathy.

Amvuttra (vutrisiran) is an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Drugs used to treat Hereditary Amyloidosis