Cystinosis is a rare disease and characterized by accumulation of cystine (a building block of proteins) inside cells. Increase in cystine damages cells and frequently forms crystals that can develop and cause issues in numerous organs and tissues. The kidneys and eyes are particularly defenseless to the damage. Muscles, thyroid, pancreas, and testicles could also be affected. Nephropathic cystinosis, intermediate cystinosis, and non-nephropathic cystinosis are the three types of cystinosis. Cystinosis disease can effect both adults and children. However, the prevalence is high in children. Juvenile nephropathic cystinosis is the most common and serious type of cystinosis disease. In cystinosis disease, a large amount of salts and glucose, bicarbonate, phosphate, potassium, and certain amino acid being excreted through the urine. This is one of the major complications of cystinosis infection (failure of kidneys) in children. In adults, cystine generally accumulates in the cornea of the eyes. Symptoms of cystinosis disease includes dehydration, vomiting, metabolic acidosis, constipation, and polyuria.
Based on treatment type, the global cystinosis disease therapeutics market can be segmented into symptomatic therapy, renal replacement therapy, and hormonal therapy. The symptomatic therapy segment is likely to expand at a rapid pace during the forecast period. The symptomatic therapy is the maintenance of fluid, electrolyte balance in the body. So, most of the doctors prefer to give dose of potassium, sodium, and phosphate substantially in addition with cystine deleting agents like Cysteamine which effectively lowers the cystine content in the cells of a patients with in short time and retain the body fluid. Moreover, the symptomatic therapy is cost effective than the other type of therapy, for this reason the symptomatic therapy segment is expected to grow at a significant pace during the forecast period. In terms of test type, the market can be classified into blood test, molecular test, urine test, slit lamp examination, and others. The blood test segment is likely to expand at a rapid pace during the forecast period.
Growing concerns about the effect of cystinosis disease in kidney, liver, and eye is one of the major factors propelling the market. Moreover, rise in awareness about mutation screening to avoid gene mutation drives the cystinosis disease therapeutics market. However, limited treatment options and lack of focus on research & development are likely to hamper the cystinosis disease therapeutics market in the next few years.
In terms of region, the global cystinosis disease therapeutic market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. Cystinosis, especially nephropathic cystinosis, is likely to affect many people in the U.S. Moreover, adoption of different types of therapy to cure cystinosis is expected to augment the cystinosis disease therapeutics market in North America. Europe is also a lucrative market for cystinosis disease therapeutics. Increase in demand for treatment and rise in patient awareness about different treatment options are anticipated to fuel the growth of the cystinosis disease therapeutics market in Europe. The cystinosis disease therapeutics market is Asia Pacific is projected to be driven by increase in population, better treatment options, and rise in demand for rapid diagnosis. Latin America and Middle East & Africa are expected to be moderate contributors to the global cystinosis disease therapeutics market.
Key players operating in the global cystinosis disease therapeutics market are Horizon Pharma plc, Mylan N.V., Novartis AG, and Gilead Sciences, among others.
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