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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. 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.

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.

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
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  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

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The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

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Lesch Nyhan Syndrome Market