News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ART001, a gene editing candidate that Accuredit Therapeutics is developing as…

Levels of a blood protein called NfL may be useful as a marker of disease activity in people with familial amyloid polyneuropathy (FAP), a…

The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary…

Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP). With the European Commission’s approval, Wainzua…

Alnylam Pharmaceuticals is planning to launch a Phase 3 clinical trial toward the end of this year to test its therapy candidate nucresiran in…

Throughout March — Amyloidosis Awareness Month — the Amyloidosis Foundation is inviting the public to join its “Light the Night for Amyloidosis” campaign, increasing…

Examining small samples of tissue from the skin and the salivary glands, those that produce saliva, increases the chances of detecting the toxic protein clumps…

Five years of treatment with Onpattro (patisiran) slowed disability progression and maintained quality of life for people with familial amyloid polyneuropathy (FAP) in…

People with familial amyloid polyneuropathy (FAP) who were given disease-modifying treatments in routine clinical care show relatively stable disease over time, but a substantial…

Researchers have uncovered previously unknown structural features of transthyretin, the protein that’s faulty in familial amyloid polyneuropathy (FAP), which could pave the way toward…