Familial Amyloid Cardiomyopathy Treatment Market Opportunity Analysis, 2018–2026

Aggregation and deposition of mutant and wild-type transthyretin protein (TTR) in heart results in familial amyloid cardiomyopathy, which typically occur after age of 60. Familial amyloid cardiomyopathy is also known as hereditary cardiac transthyretin amyloidosis or hereditary amyloid cardiomyopathy. The protein transthyretin amyloid fibrils infiltrate the myocardium that leads to diastolic dysfunction from restrictive cardiomyopathy, which eventually result to heart failure. There are several mutation in TTR which are associated with familial amyloid cardiomyopathy, which includes V1221, P24S, V20I, A45T, Gly47Val, Glu51Gly, Gln92Lys, I68L, and L111M. Most common mutation is V1221, which is substitution of isoleucine for valine at position 122 that occurs in high frequency in African-Americans, which favors the growth of familial amyloid cardiomyopathy treatment market.

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European Medicines Agency have approved drug named tafamids (Vyndaqel) for slow progression of familial amyloid cardiomyopathy in year 2013 which is expected to favor the market of familial amyloid cardiomyopathy treatment. Most of the pharmaceuticals companies are developing drugs for familial amyloid cardiomyopathy treatment, as it is associated with progressive heart failure and it is universally fatal. Symptoms for familial amyloid cardiomyopathy are weight loss, nausea, fatigue, dizziness and collapse, palpitations, abnormal heart rhythms, disrupted sleep, and angina. Genetic tests are recommended for detection of TTR gene.

Rise in amount of transthyretin protein in the body fuels the familial amyloid cardiomyopathy treatment market

Transthyretin protein is formed in the body abundantly due to inherited transthyretin gene alteration, which leads to familial amyloid cardiomyopathy as gene associated transcribes the protein. Gene alteration in body leads to alteration in sequence of body proteins. Familial amyloid cardiomyopathy are inherited by autosomal dominant allele, which can inherit the familial amyloid cardiomyopathy condition to offsprings from one of the parent. Only one copy of mutated gene is sufficient to cause the disease, which is expected to show growth in the familial amyloid cardiomyopathy market. Familial amyloid cardiomyopathy is critical disease leading to heart failure, which can favor the market. According to Amyloidosis Foundation (AF), which provides early diagnosis and advanced treatment, familial amyloid cardiomyopathy are rarely found with statistics estimating 1 per 100,000 population suffering from familial amyloid cardiomyopathy disease.

Regional growth engines of the familial amyloid cardiomyopathy treatment

On the basis of regional segmentation, the familial amyloid cardiomyopathy market can be segmented into North America, Europe, Asia Pacific, Latin America, Middle East, and Africa. According to BioMed Central journal’s published data in year March 2017, Europeans had highest number of TTR mutations, followed by East Asians, American, Central-South Asians, and Africans which favors the growth of familial amyloid cardiomyopathy market.

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The key players dominating the familial amyloid cardiomyopathy market

Familial amyloid cardiomyopathy not only affect lives of people but also affect upcoming generations, due to which manufactures are in process of releasing the drugs, as there are very few drugs approved for familial amyloid cardiomyopathy treatment. Recently in June 2017 the U.S. Food and Drug Administration (FDA), which provides accurate, science-based health information to the public, granted fast track designation to tafamidis the company’s investigational treatment for familial amyloid cardiomyopathy as there were no approved treatment in the U.S. tafamidis with trade name VYNDAQEL® was first approved in Europe. The key players operating the market are Pfizer Inc., SOM Innovation Biotech, S.L., Valeant Pharmaceuticals International, Inc., Ionis Pharmaceuticals, Inc., and AstraZeneca plc.

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