Lesch–Nyhan Syndrome (LNS) is a rare genetic disorder caused by mutation of the HPRT1 gene. Mutation of the gene leads to deficiency of the enzyme hypoxanthine-guanine phosphoribosyl transferase 1. Insufficient level of this enzyme leads to overproduction and accumulation of uric acid in bodily fluids. Accumulation of uric acid damages the kidneys, the central nervous system, the joints, and other tissues. The excessive level of uric acid may cause gouty arthritis, kidney stone, or bladder stones. It may also lead to abnormal movements of involuntary muscles such as jerking, flexing, and flailing. It may cause behavioral disturbances.
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People suffering from Lesch–Nyhan Syndrome generally cannot walk, need assistance to sit, and mostly need a wheelchair. Self-injuries such as head banging and biting is the most common and typical behavioral pattern observed in patients with Lesch–Nyhan Syndrome. The condition primarily affects men, as the HPRT1 gene is located on the X chromosome. However, a few cases are reported in women also. The rate of prevalence of LNS is around 1 in 380,000 people.
To date, no treatment has been approved for use in patients with Lesch-Nyhan Syndrome. However, the current treatment focuses on relieving of symptoms and prevention of complications. Based on treatment, the global Lesch–Nyhan Syndrome market has been segmented into medication, dietary supplements, lithotripsy, behavioral therapy, surgery, and others (including deep brain stimulation). Medication generally includes use of allopurinol, baclofen, gabapentin, carbamazepine, and pain relievers (NSAIDs). The medication segment is expected to expand at a significant pace during the forecast period, owing to promising pipeline molecules and rising incidence of Lesch-Nyhan Syndrome.
The rising incidence and prevalence of Lesch-Nyhan Syndrome, increased research and development expenditure, and growing awareness among people are factors that drive the global Lesch-Nyhan Syndrome market. Though currently available therapeutic options are effective at managing and preventing Lesch-Nyhan Syndrome, challenges of treating Lesch-Nyhan Syndrome lie in the unpredictable response of every patient, representing a major unmet need in the global Lesch-Nyhan Syndrome market. On the other hand, disappointing results of promising pipeline molecules restrain the global Lesch-Nyhan Syndrome market.
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Geographically, the global Lesch–Nyhan Syndrome market has been categorized into five regions: North America, Europe, Latin America, Asia Pacific, and Middle East & Africa. North America is anticipated to dominate the global market during the forecast period, followed by Europe. Increasing incidence and prevalence of Lesch–Nyhan Syndrome, well-established health care infrastructure, rising research and development expenditure, growing adoption of advanced treatment options, and surging health care spending in North America and Europe propel the market in these regions. The market in Asia Pacific is expected to expand at a high CAGR during the forecast period. The market in the region is likely to be driven by wide patient base, increased incidence of Lesch–Nyhan Syndrome, rising awareness, and increased government initiatives toward improving health and developing health care infrastructure in India and China during the forecast period. The Lesch–Nyhan Syndrome market in Latin America and Middle East & Africa is anticipated to expand at a significant pace during the forecast period, owing to increasing investments by key market players in these regions.
Leading players operating in the global Lesch–Nyhan Syndrome market are Psyadon Pharma Boehringer Ingelheim GmbH, AstraZeneca plc, 3SBio, Selecta Biosciences, Teijin Pharma Ltd., Phoenix Pharmacologics, GlaxoSmithKline plc., Regeneron Pharmaceuticals, Novartis AG, Merck & Co. Inc., and Savient Pharmaceuticals.