Hereditary amyloidosis (hATTR)
Investigators have found that intravenous infusion of a CRISPR-Cas9 based gene therapy decreases serum transthyretin (TTR) protein in patients with hereditary TTR…
Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and…
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually…
The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an…
Preliminary results from a landmark clinical trial suggest that CRISPR-Cas9 gene editing can be deployed directly into the body to treat disease.
The study is the…
The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the…
People living with rare diseases face a unique set of challenges, from reaching a timely and accurate diagnosis to accessing treatment. Although individual rare…
Patrick Doherty had always been very active. He trekked the Himalayas and hiked trails in Spain. But about a year and a half ago, he noticed pins and needles in his…
Amyloidosis Gelsolin Hereditary is a Facebook group that was created on 7 November, 2017.
Amyloidosis ATTRv (hATTR) - Hereditary - FAP - FAC is a Facebook group that was created on 7 September 2014.