Forecast Period 
Market Size (Base Year) 
Market Size (Forecast Year)
CAGRGlobal N-Acetylglutamate Synthase Deficiency Market, By Diagnosis (Genetic Testing, New-born Screening Others), Treatment (Medications, Alternative Treatment, Others), Drug Type (Branded, Generic), End-Users (Hospitals, Specialty Clinics, Others), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, online Pharmacies and Others) – Industry Trends and Forecast to 2030.
N-Acetylglutamate Synthase Deficiency Market Analysis and Size
N-Acetylglutamate synthase deficiency is a type of rare deficiency which happens in around 1 in 30,000 birth. This deficiency affects female and male equally. This deficiency often go unrecognized, but these disorders are underdiagnosed, making it difficult to access the frequency of urea cycle disorders in the overall population. Neurological symptoms may be life-threatening and include seizures, stupor, and coma.
Data Bridge Market Research analyses a growth rate in the N-acetylglutamate synthase deficiency market in the forecast period 2023-2030. The expected CAGR N-acetylglutamate synthase deficiency market is tend to be around 3.50% in the mentioned forecast period. The market was valued at USD 1.3 billion in 2022, and it would grow upto USD 1.71 billion by 2030. In addition to the market insights such as market value, growth rate, market segments, geographical coverage, market players, and market scenario, the market report curated by the Data Bridge Market Research team also includes in-depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.
N-Acetylglutamate Synthase Deficiency Market Scope and Segmentation
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Report Metric |
Details |
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Forecast Period |
2023 to 2030 |
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Base Year |
2022 |
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Historic Years |
2021 (Customizable to 2015 - 2020) |
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Quantitative Units |
Revenue in USD Billion, Volumes in Units, Pricing in USD |
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Segments Covered |
Diagnosis (Genetic Testing, New-born Screening Others), Treatment (Medications, Alternative Treatment, Others), Drug Type (Branded, Generic), End-Users (Hospitals, Specialty Clinics, Others), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, online Pharmacies and Others) |
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Countries Covered |
U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America |
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Market Players Covered |
Recordati Rare Diseases (Italy), Children's National Hospital (U.S.), Icahn School of Medicine at Mount Sinai (U.S.), and Boston Children's Hospital (U.S.) |
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Market Opportunities |
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Market Definition
N-acetylglutamate synthetase (NAGS) deficiency is a rare type of genetic disorder that is typically characterized by complete or partial lack of the enzyme N-acetylglutamate synthetase. Excess ammonia travels to the CNS through the blood, leading to NAGS deficiency symptoms and physical findings. This synthetase is one of the six enzymes which play a major role in the breakdown and removal of nitrogen from the body. The lack of N-acetylglutamate synthetase (NAGS) enzyme causes excessive accumulation of nitrogen in the form of ammonia in the blood.
N-Acetylglutamate Synthase Deficiency Market Dynamics
Drivers
- Increasing Treatment Developments
The treatment of NAGS deficiency aims to prevent excessive ammonia from formingaims to prevent excessive ammonia from forming or removing excessive ammonia during a hyperammonemic episode. Several long-term therapy for NAGS deficiency can be combined with dietary restrictions and the stimulation of alternate methods of converting and excreting nitrogen from the body. For instance, the U.S. FDA approved the use of Carbaglu (carbamylglutamate) tablets in 2010 to decrease blood ammonia levels in patients with NAGS deficiency. Carbaglu is manufactured by Orphan Europe. Some of the individuals who take carbamylglutamic acid may still need to follow certain dietary restrictions and may receive supplemental arginine. The initiation of carbaglu therapy has minimized the vulnerability of severe hyperammonemic episodes.
Opportunities
- Increasing Demand For Hospitals
Drug stores and hospitals are the major application segments of N-acetylglutamate synthase deficiency drugs. In 2015, the hospital segment dominated the global market with a share of more than 80% in revenue. The segment is estimated to witness major growth due to increasing healthcare expenditure and a growing number of patients suffering from life-threatening diseases
Restraints/Challenges
- Lack of Awareness
For many decades, this deficiency disease is not much known in several parts of the world. The appropriate treatment for the disease and its associated applications are unaware to the people. Thus, it hampers the market growth.
- High Cost of Treatment Process
The enormous expenditure required for its treatment processes obstructs the market growth. Numerous market players make huge investments in manufacturing new and advanced treatment procedures to faster the recovery process and in return the cost is increased.
This N-acetylglutamate synthase deficiency market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the N-acetylglutamate synthase deficiency market contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.
Global N-Acetylglutamate Synthase Deficiency Market Scope
The N-acetylglutamate synthase deficiency market is segmented on the basis of diagnosis, treatment, drug type, end-user distribution channel. The growth amongst these segments will help you analyze meagre growth segments in the industries and provide the users with a valuable market overview and market insights to help them make strategic decisions for identifying core market applications.
Diagnosis
- Genetic Testing
- New-born Screening Others
Treatment
- Medications
- Alternative Treatment
- Others
Drug Type
- Branded
- Generic
End-Users
- Hospitals
- Specialty Clinics
- Others
Distribution Channel
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacies
- Others
N-Acetylglutamate Synthase Deficiency Market Regional Analysis/Insights
The N-acetylglutamate synthase deficiency market is analyzed and market size insights and trends are provided by diagnosis, treatment, drug type, end-user distribution channel as referenced above.
The major countries covered in the N-acetylglutamate synthase deficiency market report are the U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.
North America is dominating the market ue to the presence of high healthcare expenditure and research & development.
Asia-Pacific is considered to grow over the coming years due to increased advancement in the technology as well as growing healthcare expenditure.
The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Also, the presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.
Competitive Landscape and Global N-Acetylglutamate Synthase Deficiency Market Share Analysis
The N-acetylglutamate synthase deficiency market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies' focus related to N-acetylglutamate synthase deficiency market.
Key players operating in the N-acetylglutamate synthase deficiency market include:
- Recordati Rare Diseases (Italy)
- Children's National Hospital (U.S.)
- Icahn School of Medicine at Mount Sinai (U.S.)
- Boston Children's Hospital (U.S.)
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