Onpattro (patisiran) safely and effectively helps adults with familial amyloid polyneuropathy (FAP) caused by a V122I or T60A genetic mutation keep their disease from getting worse, according to a real-world study in the U.S. The post-marketing Phase 4 study (NCT04201418) focused on patients with one of these…
Alnylam Pharmaceuticals has launched a Phase 3 clinical trial testing its experimental treatment nucresiran in adults with familial amyloid polyneuropathy (FAP), also known as hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Top-line results are expected in 2028. This update came in the company’s latest pipeline and financial report, announced…
An elderly man who developed life-threatening liver damage after receiving nexiguran ziclumeran (nex-z), an experimental gene-editing therapy, has died. The treatment was being tested for familial amyloid polyneuropathy (FAP) and the related condition ATTR amyloidosis with cardiomyopathy (ATTR-CM). The announcement came from Intellia Therapeutics, the therapy’s developer, less…
Treatment switches in people with familial amyloid polyneuropathy (FAP) are common, with Amvuttra (vutrisiran) emerging as a preferred option due to its more convenient administration route and safety profile, according to small study in Germany. “Disease progression was the primary reason for therapy switches,” researchers wrote. However, “while…
A Phase 3 clinical trial that’s testing the oral treatment acoramidis to prevent or delay disease onset is continuing to recruit adults who carry a mutation known to cause hereditary transthyretin amyloidosis (hATTR), including familial amyloid polyneuropathy (FAP), but aren’t having symptoms yet. That’s according to a recent update…
Amvuttra (vutrisiran) appears to be safe for long-term use in adults with familial amyloid polyneuropathy (FAP) and those with another form of transthyretin amyloidosis (ATTR) that causes heart damage. That’s according to an analysis of more than 700 patients who received Amvuttra in one of two Phase 3…
Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin amyloidosis (ATTR). YOLT-201 is being tested in a Phase 1/2a clinical trial (NCT06539208) that is still recruiting…
People with early-stage familial amyloid polyneuropathy (FAP) cost society more than twice as much as symptom-free carriers of disease-causing genetic mutations, largely due to higher medical needs and lost productivity at work, according to a study in Spain. “Not only early-stage [FAP] patients, but also asymptomatic carriers, are generating…
Using a small sample of tissue from skin — obtained via a skin biopsy — can help diagnose familial amyloid polyneuropathy (FAP) early by looking for the presence of disease-causing toxic amyloid deposits and measuring the number of small nerve fibers in the outermost layer of a person’s skin.
Among people carrying Val30Met (V30M), the most common cause of familial amyloid polyneuropathy (FAP), those experiencing symptoms have significantly higher blood levels of inflammation markers than those without signs of the rare inherited disease, a study showed. Symptomatic carriers were also found to have higher activity of neutrophils, a…
