Nephropathic cystinosis is the leading lysosomal storage disorder caused due to malfunctioning lysosomal membrane transfusion, and it assists as a prototype for a small group of lysosomal transportation disorders. The disease leads to intracellular growth of cysteine in all tissues and organs. Nephropathic cystinosis is an uncommon autosomal recessive disorder with lysosomal storing of the amino acid cysteine due to a deficiency in the membrane transfusion protein, cystinosis. Meanwhile the accessibility to cysteine-depleting medical therapy and the introduction of kidney transplants, to nephropathic cystinosis formerly fatal disease was transformed into a curable disease. Renal allografts and medical therapy targeting the basic metabolic deficit have transformed the natural history of nephropathic cystinosis so drastically that patients have extended a life expectancy past 50 years. Infantile nephropathic cystinosis is the most recurrent and severe form of cystinosis. Nephropathic cystinosis’s symptoms usually become deceptive within the second half of the first year of life. Precise symptoms can be severe or mild based on individual cases and the stage when management of nephropathic cystinosis is started.
Renal Fanconi syndrome and growth failure are generally the first noticeable difficulties of nephropathic cystinosis. Although infants seem regular at birth, by the age of one they frequently fall into the third percentile for weight and height. In addition, infants suffering from nephropathic cystinosis may have poor appetite, encounter feeding difficulties, and suffer from vomiting that contribute to nutritional deficit and failure to gain weight and height at the expected rate.
The global nephropathic cystinosis treatment market is segmented on the basis of therapy, end users, and geography. Based on therapy, the market is segmented into cysteine depleting therapy, symptomatic therapy, and renal transplantation. Cysteine depleting therapy can significantly lower cysteine levels inside the cells. Cysteine depleting therapy with cysteine reduces kidney damage and improves growth in children. Cysteine can expressively postpone the need for a kidney transplant. Renal Fanconi syndrome is treated with symptomatic therapy with a high intake of electrolytes and fluids to prevent extreme decrease of body dehydration. Magnesium, sodium citrate, potassium, and sodium bicarbonate may help to keep standard electrolyte stability. Acetyl cholinesterase (ACE) inhibitors are used sometimes to curtail the progress of the renal disease. Despite prompt and early treatment, patients with intermediate cystinosis and infantile ultimately develop end stage renal disease (ESRD), that requires a kidney transplant. Primarily, a suffering individual may have to endure dialysis. Dialysis is conducted through a machine that is used to ensure certain functions of the kidney. These include retaining proper levels of necessary chemicals such as potassium, and filtering waste products from the bloodstream. Based on end user, the cystinosis treatment market is segmented into hospitals, specialty clinics, and diagnostic laboratories.
On the basis of geography, the global nephropathic cystinosis treatment market is categorized into five key regions, North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. Nephropathic cystinosis is likely to affect around 2,000 patients worldwide, comprising 800 patients in Europe and 500 patients in the U.S. Cystinosis patients have hereditary malfunctioning cysteine carrier gene which outcomes in body wide cellular toxicity occurring from the abnormal accumulation of the amino acid cysteine in the lysosomes. Europe is the dominating market followed by North America in the nephropathic cystinosis treatment market. Rising patient awareness about different treatment options and increasing demand for treatment will dominate the market in Europe. The nephropathic cystinosis treatment market is expanding due to increasing population, better treatment options, and rising demand for rapid diagnosis. The global nephropathic cystinosis treatment market is expected to expand at a healthy rate in the next few years due to lack of treatment options.
The major players operating in the global nephropathic cystinosis treatment market include Horizon Pharma Plc., Mylan N.V., Novartis AG, and Gilead. Increasing competition among key players to develop new therapy for nephropathic cystinosis treatment market will drive the market globally.
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