The Urea Cycle Disorders market report covers emerging drugs, current treatment practices, market share of the individual therapies, current and forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Urea Cycle Disorders: An Overview
A Urea Cycle Disorder (UCD) is a genetic disorder that results in a deficiency of some major enzymes in the urea cycle. These enzymes are responsible for removing ammonia from the bloodstream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is changed to a compound called urea and removed from the blood.
A few of the enzymes which are involved in the Urea Cycle Disorder, collectively known as inborn errors of urea synthesis, or urea cycle enzyme defects; each is referred to by the initials of the missing enzyme: CPS1 (Carbamoyl Phosphate Synthetase), NAGS (N-Acetylglutamate Synthetase), OTC Deficiency (Ornithine Transcarbamylase), AS (Argininosuccinic Acid Synthetase (Citrullinemia)), ASL (Argininosuccinate Lyase (Argininosuccinic Aciduria)), and AG (Arginase).
Urea Cycle Disorders Treatment is a lifelong process that doesn't cure the condition, but it can effectively manage the symptoms. The child's treatment probably will involve Low protein, high-calorie diet, Medications, Amino acid supplements, and Liver transplantation.
Urea Cycle Disorders Market Key Facts
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As per the study conducted by Marshall L. Summar et al. (2013)., the estimated incidence of urea cycle disorders at 1 in 35,000 live births in the US or about 113 new patients per year.
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As per the study conducted by Mark L. Batshaw et al. (2014), the overall Incidence of urea cycle disorders is 1/35,000, with 2/3rds presenting initial symptoms after the newborn period. The mortality rate is 24% in neonatal-onset cases and 11% in late-onset cases.
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According to the literature survey, Urea Cycle Disorders can occur irrespective of sex-based differences among children. However, there may be differences in the number based on the type of the UCD.
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As per a study conducted by Susanne Nettesheim et al. (2017), titled, “Incidence, disease onset and short-term outcome in urea cycle disorders-cross-border surveillance in Germany, Austria, and Switzerland”, the estimated incidence of Urea Cycle Disorders below 16 years of age at about 1 in 52,000 live births in Germany.
Urea Cycle Disorders Market Size is expected to increase during the forecast period owing to the rise in the number of incident population of Urea Cycle Disorders patients in the 7MM. Extensive research and development activities of pharmaceutical companies for developing the therapies for Urea Cycle Disorders will also significantly impact the market.
The market outlook section of the report helps to build a detailed comprehension of the historic, current, and forecasted Urea Cycle Disorders market size by analyzing the impact of current and emerging therapies in the market. It also provides a detailed assessment of the market drivers & barriers, unmet needs, and emerging technologies.
The report gives a thorough detail of the Urea Cycle Disorders market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MoA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.
Urea Cycle Disorders Epidemiology
The epidemiology section covers insights about the historical and current Urea Cycle Disorders patient pool and forecasted trends for every seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.
Urea Cycle Disorders Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Urea Cycle Disorders market or expected to get launched in the market during the study period. The analysis covers Urea Cycle Disorders market uptake by drugs; patient uptake by therapies; and sales of each drug.
Report’s Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
The report also covers the Urea Cycle Disorders Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II, and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses the recent Development such as collaborations, acquisitions, and mergers, licensing patent details, and other information for emerging therapies.
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Urea Cycle Disorders Therapeutics Analysis
The dynamics of the Urea Cycle Disorders market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and the expected launch of emerging therapies during the forecast period.
Urea Cycle Disorders Companies:
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Acer Therapeutics
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Aeglea BioTherapeutics
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Promethera Biosciences
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Kaleido Biosciences
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Ultragenyx Pharmaceutical
And many others.
Urea Cycle Disorders Therapies covered in the report include:
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DTX301
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ACER-001
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AEB1102
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HepaStem
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KB195
And many more.
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Table of Content
1. Key Insights
2. Executive Summary
3. Urea Cycle Disorders Competitive Intelligence Analysis
4. Urea Cycle Disorders Market Overview at a Glance
5. Urea Cycle Disorders Disease Background and Overview
6. Urea Cycle Disorders Patient Journey
7. Urea Cycle Disorders Epidemiology and Patient Population
8. Urea Cycle Disorders Treatment Algorithm, Current Treatment, and Medical Practices
9. Urea Cycle Disorders Unmet Needs
10. Key Endpoints of Urea Cycle Disorders Treatment
11. Urea Cycle Disorders Marketed Products
12. Urea Cycle Disorders Emerging Therapies
13. Urea Cycle Disorders Seven Major Market Analysis
14. Attribute Analysis
15. Urea Cycle Disorders Market Outlook (7 major markets)
16. Urea Cycle Disorders Access and Reimbursement Overview
17. KOL Views on the Urea Cycle Disorders Market.
18. Urea Cycle Disorders Market Drivers
19. Urea Cycle Disorders Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The table of contents is not exhaustive; the final content may vary.
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