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…
The Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. has approved the injection therapy eplontersen — under the brand name Wainzua — for treating adults with familial amyloid polyneuropathy (FAP). Additionally, a European Union regulatory committee has recommended the treatment’s approval in the EU for adults…
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…
Gly103Arg, a mutation in the TTR gene that has been exclusively reported in Chinese people, is associated with a familial amyloid polyneuropathy-like profile marked by eye and neurological involvement, according to a case report and literature review study. All three patients from a single family carrying the mutation had…
People with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP), have significantly shorter nerve fibers in their corneas, the eye’s outermost layer, relative to healthy people, a study showed. Shorter corneal nerve fibers could be detected using a noninvasive technique known as corneal…
Long-term treatment with Onpattro (patisiran) is generally safe and results in a stabilization or lessening of neurologic impairments in people with familial amyloid polyneuropathy (FAP), according to a meta-analysis of published studies. Onpattro was used for a mean of 17.4 months (about 1.5 years) among the 503 patients in the…
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…