The parameters of muscle ultrasound, which uses sound waves to make pictures of muscles, are significantly different among people with familial amyloid polyneuropathy (FAP), those not yet showing overt disease symptoms, and healthy people. That’s according to a small study in Malaysia that suggests this noninvasive imaging scan…
A study of people carrying mutations known to cause familial amyloid polyneuropathy (FAP) found that those with disease symptoms had significantly higher levels of neurofilament light chain (NfL) — a protein that’s a marker of nerve cell damage — in their blood than those who hadn’t shown symptoms, indicating…
Abnormalities in capillaries, or very small blood vessels, and inflammation from increased blood clotting may contribute to nerve damage, or neuropathy, in advanced familial amyloid polyneuropathy (FAP), according to finding that offer new insights into the mechanisms of peripheral nerves damage in FAP, offering potential targets for developing new…
Alnylam Pharmaceuticals has taken to the road, literally, in a campaign aimed at driving home the importance of people knowing their family health history so as to better understand their risk of developing hereditary ATTR (hATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). Called…
Regulatory authorities in China have authorized YolTech Therapeutics to launch a Phase 1 clinical trial testing YOLT-201, a one-time gene-editing therapy, in people with hereditary transthyretin (hATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The approval from the National Medical Products Administration’s Center for…
For this year’s Amyloidosis Awareness Month — observed each March to call attention to these protein disorders and the people they affect — the Amyloidosis Foundation is reprising its “Light the Night for Amyloidosis” campaign. Across all 50 U.S. states, in Canada, and around the world, the goal…
Clinicians should consider familial amyloid polyneuropathy (FAP) when they see people with unexplained progressive nerve damage, or neuropathy, especially if it’s associated with bodywide symptoms or a family history of the disease, a panel of experts said in new FAP diagnosis and treatment guidelines aimed at helping U.S. doctors better…
Disease-modifying medications can significantly improve survival outcomes among people with familial amyloid polyneuropathy (FAP) no matter what age the disease begins, whereas a liver transplant only improves survival in early-onset FAP. That’s according to “Disease-Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy,” which was published in…
Running a genetic screening may help to diagnose familial amyloid polyneuropathy (FAP) among people with sensory-motor polyneuropathy — damage to many nerves involved in sensations and motor skills — and at least two symptoms suggestive of FAP. That’s according to a study in Italy that identified FAP in 10%…
New 15-year data from a global study involving more than 6,000 people with ATTR amyloidosis, an umbrella term for conditions that include familial amyloid polyneuropathy (FAP), found that nearly 1 in 4 patients experience both neurological and cardiac symptoms, showing the need, according to researchers, for improved multidisciplinary…