Understanding hATTR Amyloidosis

1. Understanding hATTR Amyloidosis (Hereditary ATTR Amyloidosis) 1) What is hATTR Amyloidosis? hATTR Amyloidosis is an inherited disease. It is characterized by the deposition of an abnormal protein called amyloid in multiple organs which causes disruption of organ tissue structure & function. It often affects the nervous system, heart & digestive tracts. Portuguese descent people are at a higher risk of inheriting this rare disease than most other people. It is a rare disease and very difficult to diagnose. (Image Source: Cleveland Clinic) 2) Symptoms of hATTR Amyloidosis: (Image Source: AKCEA Therapeutics)

2. 3) Diagnosis: People affected with hATTR Anyloidosis need an accurate & early diagnosis. Misdiagnosis for this disease is common because it's symptoms matches with any other harmful diseases. To confirm hATTR amyloidosis doctors follow some procedures such as blood tests, organ biopsies, tissue biopsies. Genetic testing can be used to confirm a mutation in the TTR gene. Once a TTR gene mutation is identified, family members of an affected individual can use this information to help determine their own risk. (Image Source: MedCity News) 4) Treatment: hATTR Amyloidosis is a rare disease & very difficult to diagnose. But early diagnosis and treatment can improve outcomes for people with hereditary ATTR amyloidosis. Genetic testing should be an important consideration for anyone with a family history of hereditary ATTR amyloidosis, or those who are experiencing symptoms of polyneuropathy. With the introduction of innovative medications specifically approved by the FDA for hereditary ATTR amyloidosis, patients now have targeted treatment options that block abnormal TTR proteins from forming, which can slow or prevent disease progression. TEGSEDI® (inotersen) is approved in the United States, Canada, and Europe for the treatment of the polyneuropathy of hereditary ATTR amyloidosis. For more information visit our website: https://hattr.ca/