Marisa Wexler, MS, senior science writer —

Long-term treatment with Vyndaqel (tafamidis) may reduce the risk of death in people with familial amyloid polyneuropathy (FAP), a study shows. The study, “Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years,” were…

Onpattro (patisiran) has been approved in Brazil to treat hereditary transthyretin amyloidosis in adults with stage 1 or stage 2 polyneuropathy, the therapy’s developer, Alnylam Pharmaceuticals, has announced. Hereditary ATTR amyloidosis, also known as familial amyloid polyneuropathy (FAP), is a genetic…

The European Commission (EC) has approved the once-daily oral medication Vyndaqel (tafamidis) for the treatment of cardiomyopathy — a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body — in adults with transthyretin…

The protein cathepsin E — known for regulating immune system cells — may be a potential biomarker for familial amyloid polyneuropathy (FAP), a study suggests. Specifically, researchers found that the protein was present at comparatively low levels in the macrophages — a type of immune cells —…

The addition of the amino acid homocysteine to the mutated transthyretin (TTR) protein found in patients with familial amyloid polyneuropathy may contribute to heart problems in these patients, a recent study suggests. Specifically, this addition destabilizes and promotes the aggregation of mutant TTR protein, but it does not affect the…

Noninvasive imaging techniques may be helpful for identifying and monitoring eye problems in familial amyloid polyneuropathy (FAP), a study shows. Titled “Multimodal retinal imaging of familial amyloid polyneuropathy,” the study was published in Ophthalmic Genetics. FAP is caused by misfolding of the protein transthyretin (TTR), which then…

Genetic sequencing can help to diagnose familial amyloid polyneruopathy (FAP) in people diagnosed with neuropathy of unknown origin — and sequencing is more reliable than biopsies for diagnosis, the results of a recent study suggest. The study, titled “Prevalence of hereditary transthyretin amyloid polyneuropathy in idiopathic…

Ambry Genetics and Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, are expanding a free genetic testing and counseling program called hATTR Compass. hATTR Compass is a no-cost program for confidential genetic testing and genetic counseling for people who are suspected of having hereditary ATTR amyloidosis, also…

Liver transplants can significantly prolong survival in familial amyloid polyneuropathy (FAP) patients, and the longer they must wait for the transplant may mean the disease will worsen after the procedure. Those findings come from the study “Liver Transplantation for Familial Amyloid Polyneuropathy (Val30Met): Long-Term Follow-up Prospective Study…