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…
Screening for toxic protein clumps using biopsies and advanced imaging techniques may help people carrying mutations that cause hereditary transthyretin amyloidosis (ATTRv) — a group of conditions that includes familial amyloid polyneuropathy (FAP) — to get an early start on disease-modifying treatment (DMT). That’s according to a study in…
The electrical signaling patterns associated with polyneuropathy, or damage to nerves outside the brain and spinal cord, vary in a first nerve conduction study given people in earlier stages of familial amyloid polyneuropathy (FAP). That’s according to a small study in Italy, which also found that signs of polyneuropathy…
Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — can separate people with symptoms of forms of hereditary ATTR amyloidosis (hATTR), including familial amyloid polyneuropathy (FAP), from those who haven’t developed symptoms yet. That’s according to a study in Italy that also showed…