News

Two Italian brothers carrying V122I, a TTR gene mutation often linked to hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), developed neurological symptoms that resembled familial…

A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin…

A newly developed second-generation transthyretin (TTR) protein stabilizer may be more effective at treating forms of TTR amyloidosis (ATTR), including familial amyloid polyneuropathy (FAP),…

Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy…

The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment…

Attralus’ AT-01 may be more sensitive than a standard imaging agent at detecting toxic transthyretin (TTR) protein clumps in the heart of people with…

More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic…

Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry mutations that cause familial…

A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the ATTR amyloidosis with cardiomyopathy (ATTR-CM),…

An international panel of patient advocates and healthcare providers has published the first set of recommendations outlining steps for the holistic care of people living…