Among familial amyloid polyneuropathy (FAP) patients carrying the most common FAP-causing mutation Val30Met, treatment with tafamidis meglumine is most effective when given at the very early stages of the disease. That’s according to the findings of a real-world study in Spain, which linked a cutoff value of 15 or…
A noninvasive test that measures axonal excitability, or the processes that underlie electrical impulses along nerve fibers, could distinguish between the early and later stages of familial amyloid polyneuropathy (FAP), a study reports. “Axonal excitability has utility to identify early and progressive nerve dysfunction in [FAP],” the researchers wrote…
Treatment with Amvuttra (vutrisiran) may ease heart damage in people with familial amyloid polyneuropathy (FAP), a new analysis of the HELIOS-A Phase 3 clinical trial suggests. “The totality of assessments in this exploratory analysis of the HELIOS-A study provides evidence of potential benefit of [Amvuttra] on cardiac [heart-related] manifestations…
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…