CHICAGO — A novel CRISPR-based in vivo gene editing therapy reduced transthyretin levels in patients with hereditary transthyretin amyloidosis with cardiomyopathy, researchers reported at the American Heart Association Scientific Sessions. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a “progressive and fatal disease,” during which amyloid deposits cause impaired
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A REDLYNCH man has spoken of how his life has been changed by a rare hereditary condition which he says has decimated his family. Vince Nicholas has transthyretin-mediated (ATTR) amyloidosis, which causes a build-up of abnormal proteins called amyloid in the body. The 61-year-old has been sharing his story to mark Rare Disease Day (Sunday, February 28).
What is Amyloidosis? Amyloidosis is the name of a group of rare and serious conditions caused by a build-up of an abnormal protein called amyloid that deposits in organs and tissues around the body. This build-up of amyloid makes it difficult for the major organs and tissues to work properly and without treatment it can lead to organ failure and death.