Carbamoyl phosphate synthetase I deficiency is a rare inherited disorder characterized by complete or partial lack of the carbamoyl phosphate synthetase (CPS) enzyme
Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment: Introduction
- Carbamoyl phosphate synthetase I deficiency is a rare inherited disorder characterized by complete or partial lack of the carbamoyl phosphate synthetase (CPS) enzyme
- This is one of five enzymes that play a key role in the breakdown and removal of nitrogen from the body, a process known as the urea cycle
- A lack of CPSI enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood
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Key Drivers of Global Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment Market
- Increase in the incidence of urea accumulation is driving the market. Moreover, the incidence of carbamoyl phosphate synthetase 1 deficiency is estimated to be between 1 in 50,000 and 1 in 300,000.
- Urea Cycle Disorder (UCD) is an assembly of rare genomic ailment, which affects approximately 3,000 - 7000 people every year in the U.S. UCD is triggered by an alteration in genes, which causes insufficiency of any of the six vital enzymes essential for the urea cycle.
- According to reports published by the CDC (Centers for Disease Control and Prevention), the anticipated incidence of UCD is 1 in 8500 births. Research findings suggest that numerous cases of UCD persist undiagnosed. In some cases, a newborn with UCD dies without a conclusive diagnosis.
- Research & development initiatives undertaken by research & academic institutes supported by pharmaceutical companies as well as governments are estimated to drive the global carbamoyl phosphate synthetase (CPS) deficiency treatment market
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Retail Pharmacies to be Highly Lucrative Segment
- Based on distribution channel, the global carbamoyl phosphate synthetase deficiency treatment market can be categorized into hospital pharmacies, retail pharmacies, online pharmacies, and others
- The retail pharmacies segment is likely to account for a major share of the global carbamoyl phosphate synthetase deficiency treatment market by 2030 due to moderate pricing of the products. Additionally, the presence of a large number of retail pharmacies, as compared to online and hospital pharmacies, is attributed to the segment’s prominent share of the global market.
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North America to Offer Significant Opportunities in Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment Market
- In terms of region, the global carbamoyl phosphate synthetase deficiency treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America is projected to expand rapidly in the next few years owing to the well-developed healthcare system, appropriate reimbursement scenario, and acceptance of latest techniques in the region
- Following North America, the carbamoyl phosphate synthetase deficiency treatment market in Europe and Asia Pacific is anticipated to expand at a rapid pace during the forecast period. Existence of well-established players, rise in investments in research and development by pharmaceutical giants, and association of pharmaceutical and biotech companies with research institutes are key drivers of the global carbamoyl phosphate synthetase deficiency treatment market.
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Key Players Operating in Global Carbamoyl Phosphate Synthetase (CPS) Deficiency Treatment Market
The global carbamoyl phosphate synthetase deficiency treatment market is consolidated, with the presence of less number of international and local players.
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