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DelveInsight Business Research LLP

DelveInsight’s”Urea Cycle Disorders Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Urea Cycle Disorders, historical and forecasted epidemiology as well as the Urea Cycle Disorders market trends in the United States, EU4 (Germany, Spain, Italy, France, and the United Kingdom) and Japan.

The Urea Cycle Disorders market report provides current treatment practices, emerging drugs, market share of individual therapies, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Urea Cycle Disorders market.

Urea Cycle Disorders Overview

Urea cycle disorders are the congenital errors of nitrogen detoxification/arginine synthesis due to defects in the urea cycle enzymes, such as carbamoylphosphate synthetase 1 (CPS1), ornithine transcarbamylase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL) and arginase 1 (ARG1), leading to respective deficiencies (abbreviated CPS1D, OTCD, ASSD, ASLD, and ARG1D). 

All the urea cycle disorders are inherited in an autosomal recessive pattern, except ornithine transcarbamoylase (OTC) deficiency, which is inherited as an X-linked trait; however, female carriers of the OTC gene can become symptomatic.

Urea Cycle Disorders Epidemiology Insights

• According to  Van Leung-Pineda (2019), the prevalence of Urea Cycle Disorders in the US is approximately 1 in 35,000, with two-thirds having symptoms in the neonatal period. The mortality rate of Urea Cycle Disorders is 24% in neonatal cases and 11% in later onset cases.

• According to Mageed G (2015), the prevalence of Urea Cycle Disorders in EU-4 was 1.89 per 100,000 in the 0-4-year-old age group, 1.63 per 100,000 in the 10-14-year-old age group, and 1.57 per 100,000 in the 15-17-year-old age group. 

• The clinical manifestations are variable. The most severe type is neonatal-onset UCD, which occurs within 28 days after birth and is characterized by vomiting, food refusal, lethargy, tachypnea, impaired consciousness, and multiorgan failure. 

• According to Batshaw et al. (2014), the mortality rate of UCD is 24% in neonatal onset cases and 11% in late-onset cases.

Click here to learn more about the Urea Cycle Disorders Market Landscape

The Report Covers the Urea Cycle Disorders Epidemiology Segmented by:

• Urea Cycle Disorders incident cases 

• Urea Cycle Disorders diagnosed cases 

• Urea Cycle Disorders treatment cases 

• Urea Cycle Disorders onset-specific cases 

Urea Cycle Disorders Market Outlook 

The Urea Cycle Disorders market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Urea Cycle Disorders market trends by analyzing the impact of current Urea Cycle Disorders therapies on the market, unmet needs, drivers and barriers, and demand for better technology. This segment gives a thorough detail of the Urea Cycle Disorders market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

According to DelveInsight, the Urea Cycle Disorders market in 7MM is expected to witness a major change in the study period 2019-2032.

 Key Companies Working in the Urea Cycle Disorders Market

• AcerTherapeutics

• Aeglea BioTherapeutics

• Ultragenyx Pharmaceutical

• And many others 

Urea Cycle Disorders Therapies Covered and Analyzed in the Report:

• ACER-001

• AEB1102

• DTX301

• And many others 

Learn more about the Key Companies and Emerging Therapies in the Urea Cycle Disorders Market

 Table of Contents 

1. Key Insights 

2. Urea Cycle Disorders Introduction 

3. Executive Summary of Urea Cycle Disorders  

4. Disease Background and Overview

5. Epidemiology and patient population

6. Urea Cycle Disorders Emerging Therapies

7. Urea Cycle Disorders Market Outlook

8. Market Access and Reimbursement of Therapies

9. Market Drivers 

10. Market Barriers 

11. Appendix

12. Report Methodology

13. DelveInsight Capabilities

14. Disclaimer

Learn about the detailed offerings of the report @ Urea Cycle Disorders Market Outlook 

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Urea Cycle Disorders Pipeline Insights

“Urea Cycle Disorders – Pipeline Insight, 2023” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Urea Cycle Disorders pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products by stage, route of administration, and molecule type. 

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