Transthyretin Amyloidosis Treatment: Introduction
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 10 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), which 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.
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The global transthyretin amyloidosis treatment market is likely to be driven by an increase in the prevalence of disease, rise in the population of people of African origin, increase in awareness, improvement in diagnostic procedures, improvement of healthcare services, rapid economic growth in developing countries, and rise in research and development activity. On the other hand, less awareness in developing economies, misdiagnosis of the disease, high cost associated with disease diagnosis and treatment, limited clinical trials, and lack of efficient medication are major factors restraining the transthyretin amyloidosis treatment market. 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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