Transthyretin amyloidosis is a rare progressive fatal disease that destroys the nerve cells controlling various bodily functions. The symptoms of the disease are similar to those of many other diseases. Moreover, patients suffering with the condition die ten years after the onset of symptoms. Therefore, timely symptom identification, diagnosis, and treatment is important.
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Transthyretin amyloidosis is caused due to the misfolding of the transthyretin (TTR) protein, which is a secondary transporter of thyroxine and retinol-binding protein. The misfolding causes protein depositions known as amyloids to form within tissues and organs. There are three types of transthyretin amyloidosis. In familial amyloid polyneuropathy (TTR-FAP) amyloids lead to damage of the nerves that control the senses, movement, and involuntary bodily functions. The second type is transthyretin amyloidosis cardiomyopathy (TTR-CM) that leads to cardiomyopathy. It causes damage to the heart’s muscles, inhibiting the ability to pump blood throughout the body. The third type is senile systemic amyloidosis (SSA), which is more prevalent in men than women.
According to the Amyloidosis Foundation, there are roughly 126 different genetic variations in ATTR, with up to 53 types of genetic variations in non-transthyretin hereditary amyloidosis diseases. According to reports published on transthyretin amyloidosis, it has been estimated that nearly 10,000, or 1.1 per 100,000 individuals in the world are living with TTR-FAP. The age group of patients suffering from the disorder is between 30 and 40 years of age. It has also been observed that TTR-CM tends to affect older males aged 65 years and above. Familial amyloid polyneuropathy (TTR-FAP) leads to 100 different types of mutations in the transthyretin gene, which leads to protein misfolding. There is only a 50% chance of transferring the mutation to the next generation from an affected parent. On the other hand, the mutation that leads to familial amyloid cardiomyopathy is generally found in individuals of African origin. Amyloidosis related to age primarily affects Caucasian men who are aged 65 years and above.
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The diagnosis of transthyretin amyloidosis is carried out through tissue biopsy, genetic testing, and imaging studies of the heart. Currently, no efficient drugs for transthyretin amyloidosis are available in the market. In order to efficiently diagnose transthyretin amyloidosis, a complete study of the family should be carried out through neurological assessment tests, which include small-fiber assessment, electrocardiogram, and laboratory tests.
The global transthyretin amyloidosis treatment market is likely to be driven by an increase in the prevalence of disease, rise in the population with African origin, increase in awareness, improvement in diagnostic procedures, improving health care services, rapid economic growth in developing countries, and rise in research and development activity. On the other hand, major restraining factors for the transthyretin amyloidosis treatment market are less awareness in developing economies, miss-diagnosis of the disease, high cost associated with disease diagnosis and treatment, limited clinical trials, and lack of efficient medication.
The global transthyretin amyloidosis treatment market is segmented into treatment type, end user, and region. The global treatment type segment of transthyretin amyloidosis treatment market is divided into surgical procedure, and medication. The end user segment in divided into hospitals, specialized clinics, academic & research institutes.
In terms of region, the global transthyretin amyloidosis treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America leads the transthyretin amyloidosis treatment market due of an increase in the African American population, rise in patient pool, increase in the number of diagnosis procedures, presence of leading manufacturers, and rapid drug development. The market in North America is followed by the market in Europe and Asia Pacific. According to the Amyloidosis Foundation, in the U.S., nearly 4,500 new cases of transthyretin amyloidosis are diagnosed every year. Asia Pacific is anticipated to provide lucrative opportunities to the transthyretin amyloidosis treatment market during the forecast period. Increase in demand for supportive care therapeutics because of an increase in health care expenditure in the region, rise in medical tourism, expansion in the pharmaceutical industry, and high rate of acceptance of new products are expected to drive the global transthyretin amyloidosis treatment market.
Major players operating in the global transthyretin amyloidosis market include Merck & Co., Pfizer Inc., AstraZeneca plc. , Prothena Corporation plc. , Ionis Pharmaceuticals, Inc., Bellus Health Inc., Alnylam Pharmaceuticals, Inc., and other prominent players.