Long-term oral treatment with tafamidis meglumine safely helped maintain nerve function, quality of life, nutritional status, and mobility in people living with familial amyloid polyneuropathy (FAP) in Japan, according to a real-world study. “This study sheds lights on the real-world safety and efficacy of tafamidis in patients who…
A protein called glial fibrillary acidic protein (GFAP) is found at higher levels in the blood of people with familial amyloid polyneuropathy (FAP), even before symptoms appear, relative to healthy people, a study finds. Also, blood levels of a protein called neurofilament light chain (NfL) were elevated only in…
Sudoscan, a test used to indirectly assess small nerve fiber damage through sweat gland function, may be a marker of disease severity and progression in people with hereditary transthyretin (ATTRv) amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP). That’s according to a small study in Italy that also showed…
Family members of people with late-onset familial amyloid polyneuropathy (FAP) may face emotional and practical challenges due to the onset of disease symptoms in late adulthood, with some seeking information and support while others avoiding discussing the disease. That’s according to interviews of eight adults who were family members…
Tafamidis meglumine safely and effectively slows disease progression in most people living with familial amyloid polyneuropathy (FAP), according to a real-world study from Brazil. The findings were in line with data from clinical trials. “The efficacy and safety of tafamidis reported in clinical trials is expandable to the…
The first adult with familial amyloid polyneuropathy (FAP) has been dosed in a Phase 3 clinical trial testing nexiguran ziclumeran (nex-z), Intellia Therapeutics’ one-time gene therapy. The trial, called MAGNITUDE-2 (NCT06672237), is recruiting up to 50 adults diagnosed with FAP, also known as hereditary transthyretin (ATTR) amyloidosis…
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 a one-time treatment for transthyretin (ATTR) amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP). Such a designation supports developing possible treatment…
Levels of a blood protein called NfL may be useful as a marker of disease activity in people with familial amyloid polyneuropathy (FAP), a new study reports — though the utility of this biomarker may vary depending on each patient’s underlying gene mutation, according to the researchers. While the…
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 transthyretin amyloidosis (ATTR-CM). Alnylam Pharmaceuticals’ Amvuttra was specifically cleared for use to reduce death, hospital stays, and urgent visits due to heart problems in…
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 can treat adults in the first two stages of FAP — stage 1, when abnormal sensations appear, but the patient is still able to…