News

MRI parameter may be new FAP biomarker, study suggests

A higher proportion of fat in the tissue that surrounds peripheral nerves, as assessed with a noninvasive MRI scan, may be a new diagnostic biomarker of familial amyloid polyneuropathy (FAP), a study showed. This measure, called intraepineurial fat fraction (ieFF), may indicate nerve fiber replacement by fat tissue and subsequent…

One-time nex-z gene-editing therapy eases FAP for two years

A one-time gene-editing therapy from Intellia Therapeutics led to a sustained, near-complete reduction in blood levels of the disease-causing transthyretin (TTR) protein in adults with familial amyloid polyneuropathy (FAP), easing symptoms or preventing them from worsening for up to two years. The data come from 36 FAP patients…

Long-term tafamidis meglumine slows FAP progression: Study

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…

Blood GFAP protein levels may help in early FAP diagnosis

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 skin test may monitor FAP progression: Study

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 late-onset FAP patients face unique challenges

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 slows FAP progression: Brazil study

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…

1st FAP patient dosed in Phase 3 trial of gene therapy nex-z

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…

ART001 gene editor named orphan drug for 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…