People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is ultimately fatal.
Most of the treatment options available to patients have involved managing the symptoms and preventing progression of the disease.