Diagnostic tests outside those considered standard for familial amyloid polyneuropathy (FAP) detected FAP in 12 of 38 people who…
Articles by Steve Bryson, PhD
A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the…
Amvuttra (vutrisiran) provided significant clinical benefits in multiple measures of quality of life and physical function in people with…
Scientists have developed a first patient-reported questionnaire on quality of life that’s specific to transthyretin amyloidosis (ATTR). Called the Transthyretin…
A newly identified mutation in the TTR gene led to various neurological and heart symptoms of ATTR amyloidosis in three…
Adults with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), lived longer after…
Caring for a person with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP),…
Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people…
Older age and worse disease were significantly associated with cognitive impairments in people with familial amyloid polyneuropathy (FAP) who…
Eplontersen reduced transthyretin (TTR) protein levels, eased disease progression, and improved quality of life in adults with familial…