News

Early, late FAP stages distinguished with noninvasive nerve fiber test

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…

Heart damage markers fell for FAP patients on Amvuttra: Phase 3 trial

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…

Muscle ultrasound may help monitor FAP severity

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…

New Alnylam program focuses on need to know family health history

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…

China regulators OK Phase 1 trial of gene-editing therapy YOLT-201

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…

New FAP treatment guidelines can help US doctors: Expert panel

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…