Akcea Therapeutics Inc.
Akcea Therapeutics Inc., is a North American biopharmaceutical company. The Company develops and commercializes drugs for the treatment of patients with cardiometabolic diseases caused by lipid disorders. Our priority is to drive clinical program execution, understand patient and physician needs, prepare the market, create market access, and commercialize our products on a global basis.
Treatment Options For hATTR Amyloidosis - Akcea Therapeutics Canada. In this article, we are going to discuss two of the most recent and promising treatment options for hATTR amyloidosis.
Patisiran (Onpattro® 2 mg per ml concentrate for the preparation of an infusion solution, Alnylam Pharmaceuticals) and Inotersen (Tegsedi® 284 mg injection solution in a pre-filled syringe, Ionis) are the two new active ingredients for the treatment of patients with hereditary transthyretin-mediated amyloidosis. Around 50,000 people worldwide are affected by hATTR Amyloidosis. The disease is progressive and can lead to morbidity and disability, and potentially death, within 2 to 15 years. Often, hATTR is underdiagnosed or misdiagnosed. The TTR stabilizer Tafamidis (Vyndaqel®) has been available as a therapeutic option so far. Patisiran: The First RNAi Therapeutic Agent The principle of action of the two new drugs is different, but in the end, it comes down to both of them blocking the formation of TTR.
Our Commitment - Akcea Therapeutics. Understanding the Stages of hATTR Amyloidosis - Akcea Therapeutics Canada. Amyloidosis is a term used to define a group of diseases characterized by extracellular deposition of fibrillar proteinaceous substances called amyloid that have common morphological, structural and staining properties but with variable protein composition.
The nomenclature of different forms of amyloid is done by putting the alphabet A (A for amyloid), followed by the suffix derived from the name of the specific protein constituting amyloid of that type. For instance, ATTR stands for Amyloid Transthyretin (TTR). It is a serum protein synthesized in the liver that facilitates the transport of thyroxine and retinol. Hereditary polyneuropathy (ATTR) is an autosomal dominant, multi-system, progressive and life-threatening disease in which amyloid gets deposited in the peripheral and autonomic nerves, which gradually results in muscular weakness, pain, paresthesia and cardiomyopathy.
About 60 types of such mutations have been defined to date. Coutinho Staging System PND l PND ll PND lll-A PND lV. What is the life expectancy of someone with amyloidosis? Potential Treatment of Hattr Amyloidosis. Slide 1: Potential Treatment of hATTR Amyloidosis slide 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 amyloidosis since 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. Slide 1: How to Talk To Your Family and Relatives if hATTR Amyloidosis Runs in Your Genes. Whether you have relatives with hATTR amyloidosis or you have been diagnosed as the only one in the family, talking to your family is an important first step in raising awareness.
Educating your family about hATTR amyloidosis can help you and your family make informed, health-conscious decisions about your future. It’s time to start the conversation. hATTR amyloidosis is a serious illness and you may be unsure how to start the conversation with loved ones. Initiating the Conversion: ● Learn the facts The more you know about hATTR amyloidosis, the better you will be able to answer your loved one’s questions and the better prepared you will be to deal with your illness.
Tegsedi – Akcea Therapeutics. FCS - Everything You Need To Know. What are some potential treatments of Amyloidosis? Our Approach – Akcea Therapeutics. HATTR Amyloidosis - About, Symptoms, Causes, Treatment & Risk Factors. What are the symptoms of familial chylomicronemia syndrome? What are the diagnosis of amyloidosis? Biopharmaceutical Company Canada - Akcea Therapeutics Inc. Tegsedi - Inotersen Injection for HATTR Amyloidosis. Tegsedi (inoterseninjection)What is TEGSEDI?
• TEGSEDI™ (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). • This medicine can help reduce symptoms such as pain, numbness, tingling, abnormal heartbeats, diarrhea, constipation, weakness, and problems with movement in your arms or legs.What is TEGSEDI used for? • TEGSEDI is used for treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). • In hATTR, a change or mutation in the gene alters the structure of the TTR protein causing it to fold into an unusual shape or clump together and build up in the body. This buildup of TTR can stop the organs in your body from working properly.How does TEGSEDI work?
• TEGSEDI belongs to a group of medicines called anti-sense oligonucleotides (ASO). What causes hereditary ATTR amyloidosis? Tegsedi – hATTR Amyloidosis Treatment - Akcea Therapeutics. Familial Chylomicronemia Syndrome (FCS) - About, Causes, Sign & Symptoms. Hereditary transthyretin (hATTR) amyloidosis – Akcea Therapeutics. Biopharmaceutical Company Canada - Akcea Therapeutics Canada.