Two trials testing nexiguran ziclumeran (nex-z), a gene-editing therapy for familial amyloid polyneuropathy (FAP) and the related condition ATTR…
Articles by Marisa Wexler, MS
Levels of a blood protein called NfL may be useful as a marker of disease activity in people with…
Five years of treatment with Onpattro (patisiran) slowed disability progression and maintained quality of life for people with…
Researchers have uncovered previously unknown structural features of transthyretin, the protein that’s faulty in familial amyloid polyneuropathy (FAP), which…
Onpattro (patisiran), an approved treatment for familial amyloid polyneuropathy (FAP), is now listed on Australia’s Pharmaceutical Benefits Scheme…
Treatment with Amvuttra (vutrisiran) may ease heart damage in people with familial amyloid polyneuropathy (FAP), a new analysis of…
Disease-modifying medications can significantly improve survival outcomes among people with familial amyloid polyneuropathy (FAP) no matter what age the disease…
The U.S. Food and Drug Administration (FDA) has approved eplontersen for treating familial amyloid polyneuropathy (FAP). The therapy, which…
Screening for toxic protein clumps using biopsies and advanced imaging techniques may help people carrying mutations that cause hereditary transthyretin…
More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the…