Prothena Biosciences
This seminar will provide information on the treatment and management of ATTR amyloidosis, as well as a discussion of future directions for research and treatment.…
In February last year, Doherty - now about age 65 - began to experience the same early breathing symptoms his father had had. As an avid hiker who has trekked the…
The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of…
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…