Potential Treatment of hATTR Amyloidosis - Akcea Canada

1. Potential Treatment of hATTR Amyloidosis

2. There are different types of amyloidosis and grouping them all into one single disease will not get you anywhere, and definitely not to the right answer to your question. I have some knowledge on hereditary amyloidosissince it runs in my father’s family (fortunately, I am not showing any of its symptoms yet). More commonly known as hATTR amyloidosis, although it’s already a very rare disease, to begin with, it’s a part of a group of diseases that develop due to the buildup of amyloid deposits in the body. Only about 50,000 people around the world have it, and this rarity is what makes it so difficult to diagnose. Additionally, misdiagnosis of this disease is very common and it can often take patients several years from a symptom onset to get the right diagnosis and to begin treatment. People of Portuguese descent are at a higher risk of inheriting this rare disease than others. Genetic testing is a crucial step in getting yourself correctly diagnosed. If anyone in your family/close relatives has been diagnosed with hATTR amyloidosis, I would highly recommend getting diagnosed as soon as possible since it’s a very progressive disease and can be life-threatening. Early diagnosis can help suppress your situation significantly. An individualized treatment plan developed in collaboration by an amyloidosis specialist and other healthcare professionals has shown to benefit patients to a great degree. Also, medications are quite beneficial for symptom management. I hope this helps. For instance, Tegsedi® by AKCEA, an inotersen solution for subcutaneous injection, is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).