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…
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…
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…
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…
Applying a fluorescent green tracer called AMDX-9101 during a routine eye exam may enable the detection of the toxic transthyretin protein deposits that cause symptoms of transthyretin amyloidosis (ATTR), a group of conditions that include familial amyloid polyneuropathy (FAP). It’s too early to know if the approach would work…
Nearly every fifth adult with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has renal tubular acidosis (RTA) — a problem that occurs when the kidneys fail to remove acids from the blood into the urine. That’s according to a new study from…
Young adults at risk for familial amyloid polyneuropathy (FAP) face unique psychological and social challenges, and likely would benefit from tailored healthcare support when undergoing pre-symptomatic testing for the rare disease. That’s according to a new study by researchers in Portugal, who conducted interviews with more than a dozen…
The first patient has been dosed in a China-based Phase 1/2a clinical trial testing YOLT-201, Yoltech Therapeutics’ one-time gene-editing therapy, in people with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP). Two weeks after receiving the investigational therapy, which was given as…
The oral medication tafamidis meglumine appears to be well tolerated by adults with familial amyloid polyneuropathy (FAP) in France, where the treatment is approved under the brand name Vyndaqel and is often started at an early stage of the disease, a real-world study finds. Easy access to a…