News

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…

Medications improve FAP survival regardless of patients’ age at onset

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…

1 in 4 with ATTR amyloidosis have neurological and heart symptoms

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…

Unconventional tests detect FAP in 12 of 38 asymptomatic carriers

Diagnostic tests outside those considered standard for familial amyloid polyneuropathy (FAP) detected FAP in 12 of 38 people who carried disease-causing mutations but were asymptomatic, or without evident FAP symptoms, a study reported. The assessment methods, which identified FAP features when standard tests did not, included modified nerve…