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…
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 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…
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…