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…
An ultrasound of the peripheral nerves could help monitor disease progression in people with early-stage familial amyloid polyneuropathy (FAP) who carry V30M, the most common disease-causing mutation. That’s according to a study in Sweden that also showed that while about half of patients analyzed showed no signs of peripheral…
Two Italian brothers carrying V122I, a TTR gene mutation often linked to hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), developed neurological symptoms that resembled familial amyloid polyneuropathy (FAP), with little or no heart involvement, a study shows. The cases add to the growing number of studies reporting symptoms of nerve…
A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…
A newly developed second-generation transthyretin (TTR) protein stabilizer may be more effective at treating forms of TTR amyloidosis (ATTR), including familial amyloid polyneuropathy (FAP), than one already tested in patients, scientists in Spain announced. Called PITB, the molecule was seen to be better than tolcapone — a TTR…
Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent…