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…
The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary transthyretin amyloidosis (ATTR-CM). Alnylam Pharmaceuticals’ Amvuttra was specifically cleared for use to reduce death, hospital stays, and urgent visits due to heart problems in…
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…
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…
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…
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…