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…
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…
Running a genetic screening may help to diagnose familial amyloid polyneuropathy (FAP) among people with sensory-motor polyneuropathy — damage to many nerves involved in sensations and motor skills — and at least two symptoms suggestive of FAP. That’s according to a study in Italy that identified FAP in 10%…
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…
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…
A few weeks after its approval in the U.S., Wainua (eplontersen) will now be available to adults with familial amyloid polyneuropathy (FAP). Orsini was selected as the exclusive specialty pharmacy partner to provide Wainua, which was co-developed by Ionis Pharmaceuticals and AstraZeneca. The medication will also be made available through integrated…
The U.S. Food and Drug Administration (FDA) has approved eplontersen for treating familial amyloid polyneuropathy (FAP). The therapy, which will be sold under the brand name Wainua by its co-developers Ionis Pharmaceuticals and AstraZeneca, is specifically indicated for adults with the genetic disease. In a previous Phase…
A little more than a year of treatment with eplontersen was found to stabilize or even improve the heart’s structure and function in people with familial amyloid polyneuropathy (FAP) who also were experiencing symptoms of heart disease, known as cardiomyopathy. That’s according to an exploratory analysis of data…