Throughout March — Amyloidosis Awareness Month — the Amyloidosis Foundation is inviting the public to join its “Light the Night for Amyloidosis” campaign, increasing awareness about a group of rare diseases that includes familial amyloid polyneuropathy (FAP). People are asked to brighten the entrance or features of their…
Examining small samples of tissue from the skin and the salivary glands, those that produce saliva, increases the chances of detecting the toxic protein clumps that cause familial amyloid polyneuropathy (FAP) early in the disease’s course, a study reported. Its findings suggest that analyzing both these tissues, whose collection…
Five years of treatment with Onpattro (patisiran) slowed disability progression and maintained quality of life for people with familial amyloid polyneuropathy (FAP) in a long-term clinical trial, a study shows. Although Onpattro can help delay FAP progression, “patients tend not to recover function that is lost before starting…
People with familial amyloid polyneuropathy (FAP) who were given disease-modifying treatments in routine clinical care show relatively stable disease over time, but a substantial proportion still showed signs of disease progression throughout follow-up, a real-world study in France shows. “In routine care, the overall population of patients remains stable,”…
Researchers have uncovered previously unknown structural features of transthyretin, the protein that’s faulty in familial amyloid polyneuropathy (FAP), which could pave the way toward new treatment strategies. “We’ve unveiled a molecular complexity that has been hidden from researchers for decades, which enables us to design better medicines to stabilize…
People with familial amyloid polyneuropathy (FAP) have fewer autonomic symptoms caused by damage to the nerves that control involuntary bodily functions after more than 1.5 years of treatment with Wainua (eplontersen) compared with a placebo. That’s according to a new secondary analysis from the Phase 3 NEURO-TTRansform…
A single infusion of YOLT-201, Yoltech Therapeutics’ investigational gene-editing therapy, appears to be safe and to effectively reduce blood levels of the disease-driving TTR protein in people with familial amyloid polyneuropathy (FAP). That’s according to preliminary data from six FAP patients given the therapy in the initial,…
A report on a 64-year-old man with familial amyloid polyneuropathy (FAP) may be the first to describe the buildup of disease-causing toxic clumps called amyloid deposits inside Schwann cells, specialized nervous system cells. Schwann cells wrap around nerve fibers, covering them with myelin, a fat-rich protective layer…
Throughout 2024, FAP News Today informed our readers about the latest developments in treatment and clinical trials for familial amyloid polyneuropathy (FAP). Here is a list of the top 10 most read stories we published in 2024, along with a brief summary. We look forward to continuing to…
Problems with memory and problem-solving in people with hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), associated with a thinning in specific brain regions, according to a small brain imaging study. “The reasons for cognitive involvement in ATTRv are still a subject of…