Albany, NY -- (SBWIRE) -- 08/02/2019 -- Urea Cycle Disorders Market Insights, Epidemiology and Market Forecast-2028
1. Urea Cycle Disorders incidence is below 16 years of age at about 1 in 52,000 live births in Germany.
2. The urea cycle disorders are inherited in an autosomal recessive way.
3. Urea Cycle Disorders incidence for the United States is predicted to be 1 urea cycle disorder patient for every 35,000 births presenting about 113 new patients per year across all age groups
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Key benefits
1. Urea Cycle Disorders market report covers a descriptive overview and comprehensive insight of the Urea Cycle Disorders epidemiology and Urea Cycle Disorders market in the 7 MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
2. Urea Cycle Disorders market report provides insights into the current and emerging therapies.
3. Urea Cycle Disorders market report provides a global historical and forecasted market covering drug outreach in 7 MM.
4. Urea Cycle Disorders market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Urea Cycle Disorders market.
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"Urea Cycle Disorders can occur irrespective of sex-based differences among children."
Recent advances in Urea Cycle Disorders treatment of inborn errors of urea synthesis have significantly decreased the mortality rate. Urea Cycle Disorders treatment consists of dietary management to limit ammonia production in conjunction with medications and supplements which provide alternative pathways for the removal of ammonia from the bloodstream. The goal is to correct biochemical abnormalities and make sure adequate nutritional intake. Urea Cycle Disorders treatment includes compounds that raises nitrogen waste removal. These compounds are converted from nitrogen to products other than urea that is excreted; so, the load on the urea cycle is decreased. The first compounds that were used: sodium benzoate and arginine. Afterwards, phenylacetate was used, which has now been replaced by phenylbutyrate.
In the current Urea Cycle Disorders market scenario, recommended therapies to possibly slow the progression of the disease include sodium benzoate, sodium phenylacetate, Glycerol phenylbutyrate, sodium phenylbutyrate, and Antiemetic agents. These drugs decrease blood ammonia concentrations by conjugation reactions including amino acids acylation. Sodium phenylbutyrate is a prodrug and is metabolized to phenylacetate. Phenylacetate then conjugates with glutamine for phenylacetylglutamine, which is then excreted by the kidneys. Glycerol phenylbutyrate is a nitrogen-binding agent for chronic management of adult and pediatric patients with urea cycle disorders which cannot be managed by dietary protein restriction and amino acid supplementation alone. Carglumic acid is a structural analogue of N -acetylglutamate, which enters cells and enables activation of CPS I in vivo & prevents hyperammonemia by converting ammonia into urea. It is more resistant to enzymatic degradation by hydrolysis compared with N -acetylglutamate. Antiemetic agents such as Ondansetron hydrochloride, Granisetron and Palonosetron control nausea, and vomiting associated with IV administration of sodium benzoate and phenylacetate.
Several Urea Cycle Disorders treatments can be effective; however, there are still several unmet needs in the management of the disease. Alternative pathway therapy has limited effectiveness in preventing hyperammonemia and is combined with effective dietary management. Therefore in children with a neonatal-onset disease or those with inferior metabolic control, liver transplantation is also considered. So, there is an immediate need for research and development activity for the development of Urea Cycle Disorders therapies that can overcome the limitations of existing therapies.
The launch of the emerging therapies is expected to significantly impact Urea Cycle Disorders treatment scenario in the upcoming years:-
Drugs covered
1. ACER-001
2. SYNB1020
3. DTX301
4. AEB1102
And many others
The key players in Urea Cycle Disorders market are:
1. Acer Therapeutics
2. Aeglea BioTherapeutics
3. Synlogic
4. Ultragenyx Pharmaceutical
And many others
Table of contents
1. Report Introduction
2. Urea Cycle Disorders Market Overview at a Glance
3. Urea Cycle Disorders Disease Background and Overview
4. Urea Cycle Disorders Epidemiology and Patient Population
5. Urea Cycle Disorders Epidemiology by Countries (2017-2028)
5.1. United States- Epidemiology (2017-2028)
5.2. EU-5 - Epidemiology (2017-2028)
5.3. Germany-Epidemiology (2017-2028)
5.4. France-Epidemiology (2017-2028)
5.5. Italy-Epidemiology (2017-2028)
5.6. Spain-Epidemiology (2017-2028)
5.7. United Kingdom-Epidemiology (2017-2028)
5.8. Japan-Epidemiology (2017-2028)
6. Urea Cycle Disorders Treatments & Medical Practices
7. Urea Cycle Disorders Marketed Drugs
7.1. Ammonul: Ucyclyd Pharma
7.2. Buphenyl: Horizon Pharma
7.3. Ravicti: Hyperion Therapeutics
8. Urea Cycle Disorders Emerging Therapies
8.1. Key Cross Competition
8.2. ACER-001: Acer Therapeutics
8.3. SYNB1020: Synlogic
9. Urea Cycle Disorders Market Size
10. 7MM: Country-Wise Market Analysis
10.1. United States Market Size
10.2. Germany Market Size
10.3. France Market Size
10.4. Italy Market Size
10.5. Spain Market Size
10.6. United Kingdom Market Size
11. Market Drivers
12. Market Barriers
13. Urea Cycle Disorders Report Methodology
14. DelveInsight Capabilities
15. About DelveInsight
About DelveInsight
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