A skin sample might aid physicians in diagnosing patients with familial amyloid polyneuropathy (FAP) caused by mutations in the TTR gene, according to a study that showed that amyloid deposits, which cause nerve disease in these patients, are also present in the skin. In addition to sparing patients more invasive…
The investigative drug inotersen was shown to significantly reduce neurological progression and improve quality of life in patients with familial amyloid polyneuropathy (FAP) – also referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). These findings resulted from the Phase 3 NEURO-TTR study (NCT01737398), and were subject of a…
The age at which symptoms of familial amyloid polyneuropathy (FAP) start showing up may be influenced by other genetic factors, according to a study that assessed genetic variation in a large number of patients.