News

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…

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…

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…

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…

Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through…

The Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. has approved the injection therapy eplontersen — under the brand name Wainzua —…

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…

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…

People with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP), have significantly shorter nerve fibers in their corneas,…

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),…