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…
Two trials testing nexiguran ziclumeran (nex-z), a gene-editing therapy for familial amyloid polyneuropathy (FAP) and the related condition ATTR amyloidosis with cardiomyopathy (ATTR-CM), are on hold after a participant developed potentially life-threatening liver damage. The participant received the therapy on Sept. 30 as part of the Phase 3…
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…
A single dose of gene-editing therapy nexiguran ziclumeran (nex-z) leads to deep reductions in blood levels of the disease-causing transthyretin (TTR) protein in adults with familial amyloid polyneuropathy for up to three years. That’s according to longer-term data from a Phase 1 clinical trial (NCT04601051). The trial also…
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…
There is a high prevalence of the Val30Met (V30M) TTR gene mutation — the most common mutation associated with familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin amyloidosis (ATTRv) — in Spain’s Balearic Islands, according to a new report by scientists in the region. The scientists believe…
