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…
A single dose of nucresiran, an experimental therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis, safely resulted in significant and sustained reductions in blood levels of the disease-driving transthyretin (TTR) protein in healthy people. That’s according to interim results from an ongoing, placebo-controlled Phase 1…
A mutation of the TTR gene that’s been exclusively reported in Chinese people causes an eye condition called vitreous opacity that can be successfully managed with eye surgery, but eventually recurs in nearly all cases after a mean of five years, a study shows. After surgery, which is called…
One year after a single dose of Intellia Therapeutics’ experimental gene-editing therapy NTLA-2001, blood levels of the disease-driving transthyretin (TTR) protein dropped by a mean of 91% in people with familial amyloid polyneuropathy (FAP). Available data at two years showed sustained TTR level reductions, which were accompanied…
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to AT-02, Attralus’ investigational candidate for the treatment of transthyretin amyloidosis (ATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP). Orphan drug status supports the development of potential treatments for rare diseases, those…
Diflunisal is used off-label to treat familial amyloid polyneuropathy (FAP), but it may be as safe and effective as tafamidis free acid, which is approved in the U.S. under the brand name Vyndamax for treating a FAP-related condition, a study in Taiwan suggests. Nearly all the evaluated FAP patients…
Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through public drug plans, the therapy’s developer said. Amvuttra is approved in the country for adult FAP patients with stage 1 or stage 2 polyneuropathy or…