Novel gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy miragenews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from miragenews.com Daily Mail and Mail on Sunday newspapers.
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.