Marisa Wexler, MS, senior science writer —

Treatment with Onpattro (patisiran) or Amvuttra (vutrisiran) leads to a reduction in blood levels of neurofilament light chain (NfL), a nerve cell damage marker, among people with familial amyloid polyneuropathy (FAP), according to a new analysis of clinical trial data. Findings suggest that patients with a greater…

People with hereditary transthyretin amyloidosis (hATTR) who are treated with a combination of therapies appear to have better health outcomes than those given only one type of medication. That’s according to new findings presented at the 75th American Academy of Neurology (AAN) annual meeting, being held April 22-27 in Boston…

In the U.S., one-third of people with hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are diagnosed with carpal tunnel syndrome years before a hATTR diagnosis, a study indicated. This finding suggests that a history of carpal tunnel, a condition that affects…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Ionis Pharmaceuticals seeking approval of eplontersen in treating familial amyloid polyneuropathy (FAP). The agency is expected to announce its decision by Dec. 22, according to Ionis, which is co-developing eplontersen alongside…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Alnylam Pharmaceuticals seeking the approval of Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. According to the company, the FDA is expected to announce a decision by Oct. 8. Alnylam submitted…

Most people with late-onset familial amyloid polyneuropathy (FAP) experience pain as a result of nerve damage, referred to as neuropathic pain, a recent study highlights. Findings suggest that neuropathic pain is linked to worse life quality, and many patients do not experience relief from this type of pain even…

England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available through the country’s National Health Service (NHS) for treating familial amyloid polyneuropathy (FAP). Amvuttra is one of the first rare disease treatments to get the green light from NICE…

Eye involvement can become common over time in people with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), a study from Spain reported. Researchers also noted that symptoms affecting the eyes are often slower to develop than others due to this disease. As…

Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve Onpattro (patisiran) for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. Onpattro already is approved to treat familial amyloid polyneuropathy (FAP), a genetic disorder in which toxic tangles…

One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). That is according to data shared by NTLA-2001’s co-developer, Intellia Therapeutics, at the American Heart Association…