News

Early-stage FAP brings heavy economic burden for society

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…

Nex-z trial for FAP to complete enrollment earlier than expected

A Phase 3 clinical trial testing Intellia Therapeutics’ gene-editing therapy nexiguran ziclumeran (nex-z) in adults with familial amyloid neuropathy (FAP) is ahead of its planned enrollment schedule, with recruitment now expected to be completed in the first half of 2026. That’s according to a press release from…

Medications show promise for treating kidney disease in ATTRv

Kidney disease is an underrecognized complication of hereditary transthyretin amyloidosis (ATTRv) — a group of diseases that includes familial amyloid polyneuropathy (FAP) — but may be effectively controlled with a class of medications known as siRNA therapies, among them Onpattro (patisiran) and Amvuttra (vutrisiran). That’s according to an observational study,…

Neuropathy linked with TTR mutation V142I: Study

Neuropathy, or nerve-related pain, is more prevalent than previously reported in people with the V142I mutation of hereditary transthyretin amyloidosis (ATTRv), a study found. Familial amyloid polyneuropathy (FAP), a clinical form of ATTRv, typically involves progressive nerve damage. V142I, the most common ATTRv mutation in the U.S., has traditionally…

Inflammation markers linked to symptoms in Val30Met-related FAP

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…

New MRI technique may help detect nerve damage in FAP: Study

A specialized MRI technique known as the variable echo time (vTE) sequence may help identify structural changes in nerves linked to damage in individuals with familial amyloid polyneuropathy (FAP), according to a recent study. Additionally, a parameter of the vTE MRI sequence called short T2-weighted imaging was identified as…

Amvuttra gets EU approval to treat adults with ATTR-CM

The European Commission has expanded the indication of Amvuttra (vutrisiran) to include adults with ATTR amyloidosis with cardiomyopathy, or heart damage (ATTR-CM), regardless of whether they carry disease-causing mutations. “Estimates show approximately 100,000 people are affected by ATTR amyloidosis across Europe, most with cardiomyopathy, so this approval marks a…