hATTR-PN

Family members of people with late-onset familial amyloid polyneuropathy (FAP) may face emotional and practical challenges due to the onset of disease symptoms in late adulthood, with some seeking information and support while others avoiding discussing the disease. That’s according to interviews of eight adults who were family members…

Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation. Historically, New Zealand has maintained some of the strictest gene technology regulations globally. Currently, genetic modification is heavily regulated under the Hazardous Substances and New Organisms Act…

Tafamidis meglumine safely and effectively slows disease progression in most people living with familial amyloid polyneuropathy (FAP), according to a real-world study from Brazil. The findings were in line with data from clinical trials. “The efficacy and safety of tafamidis reported in clinical trials is expandable to the…

The first adult with familial amyloid polyneuropathy (FAP) has been dosed in a Phase 3 clinical trial testing nexiguran ziclumeran (nex-z), Intellia Therapeutics’ one-time gene therapy. The trial, called MAGNITUDE-2 (NCT06672237), is recruiting up to 50 adults diagnosed with FAP, also known as hereditary transthyretin (ATTR) amyloidosis…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ART001, a gene editing candidate that Accuredit Therapeutics is developing as a one-time treatment for transthyretin (ATTR) amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP). Such a designation supports developing possible treatment…

Levels of a blood protein called NfL may be useful as a marker of disease activity in people with familial amyloid polyneuropathy (FAP), a new study reports — though the utility of this biomarker may vary depending on each patient’s underlying gene mutation, according to the researchers. While the…

Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP). With the European Commission’s approval, Wainzua can treat adults in the first two stages of FAP — stage 1, when abnormal sensations appear, but the patient is still able to…

Through my roles as a caregiver for my late husband, Aubrey, and as an amyloidosis patient advocate, I’ve been transformed. I once assumed that my life would involve being a wife and stay-at-home mother, with a happy home and a couple of pets to boot. But I became disgruntled…

Alnylam Pharmaceuticals is planning to launch a Phase 3 clinical trial toward the end of this year to test its therapy candidate nucresiran in people with familial amyloid polyneuropathy (FAP) — though details on the trial’s design are still being discussed with global regulators. That’s according to pipeline…

The daily life of a familial amyloid polyneuropathy (FAP) caregiver isn’t always one of set routines. With the nature of the ailment and its painful peripheral neuropathy symptoms, there will be days when new issues arise or symptoms become more severe. Some of these problems may be unforeseen,…