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hATTR is a rare condition caused by abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in the body’s tissues and organs. It progresses rapidly and can be fatal, with a median survival of just 4.7 years from diagnosis.NEXT ARTICLE
Most human diseases are caused by production of abnormal proteins or malfunctioning proteins. Antisense therapy involves inhibiting production of these proteins. When a gene is known to cause a specific disease and the genetic sequence ...