News

Combination therapy may be safe in FAP patients: Study

Combination therapy for hereditary transthyretin amyloidosis (hATTR), a group of conditions including familial amyloid polyneuropathy (FAP), appears to be safe, according to a retrospective study, but because its effects “on neurological symptoms could not be assessed,” more studies are needed to confirm its benefits, the researchers wrote. Most of…

Young adults at risk of FAP may need tailored healthcare support

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…

Heart damage-linked gene caused FAP-like symptoms: Case report

A man carrying p.Val142Ile, a TTR gene mutation often linked to a condition related to familial amyloid polyneuropathy (FAP) but causing heart damage instead of nerve damage, developed FAP-like neurological symptoms and no heart involvement, a case report showed. “The particularly unusual [genetic profile-clinical profile] association distinguishes this case…

First hATTR patient dosed in Phase 1/2a trial of gene-editing therapy

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…

Second NTLA-2001 dose safely lowers TTR levels in FAP patients

A second, therapeutic dose of Intellia Therapeutics‘ experimental gene-editing therapy NTLA-2001 safely and effectively reduced levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP). That’s according to data from the three FAP patients first treated with a suboptimal, low dose of NTLA-2001 who…