Adults who carry the Val30Met, or V30M, mutation — the most common cause of early-onset hereditary transthyretin amyloidosis with…
Articles by Margarida Maia, PhD
Polyneuropathy, or widespread nerve damage, occurs in both hereditary and wild-type transthyretin amyloidosis (ATTR), but the hereditary form, which includes…
Onpattro (patisiran) safely and effectively helps adults with familial amyloid polyneuropathy (FAP) caused by a V122I or T60A…
Alnylam Pharmaceuticals has launched a Phase 3 clinical trial testing its experimental treatment nucresiran in adults with familial…
An elderly man who developed life-threatening liver damage after receiving nexiguran ziclumeran (nex-z), an experimental gene-editing therapy, has died. The…
Treatment switches in people with familial amyloid polyneuropathy (FAP) are common, with Amvuttra (vutrisiran) emerging as a preferred…
A Phase 3 clinical trial that’s testing the oral treatment acoramidis to prevent or delay disease onset is continuing to…
Amvuttra (vutrisiran) appears to be safe for long-term use in adults with familial amyloid polyneuropathy (FAP) and those…
Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing…
People with early-stage familial amyloid polyneuropathy (FAP) cost society more than twice as much as symptom-free carriers of disease-causing…