Urea Cycle Disorders Market Share & Size, Market Trends and Forecast till 2030

 

Urea Cycle Disorders Market

 

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 blood stream. 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.

 

Normally, the urea is removed from the body through the urine. In urea cycle disorders, nitrogen builds up in the blood in the form of ammonia, a highly toxic substance, resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma, and/or death. The onset and severity of urea cycle disorders is highly variable. The severity correlates with the amount of urea cycle enzyme function.

 

DelveInsight's "Urea Cycle Disorders - Market Insights, Epidemiology, and Market Forecast-2030" 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, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

 

 

Geography Covered

·         The United States

·         EU5 (Germany, France, Italy, Spain, and the United Kingdom)

·         Japan

 

Study Period: 2017-2030

 

Urea Cycle Disorders Disease Understanding and Treatment Algorithm

 

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).

 

In children with severe UCD, the symptoms will develop within the first 24 hours of life. Symptoms in children with mild or moderate UCD, who do not show symptoms until early childhood, may include: Vomiting, nausea, Mental confusion or hyperactive behavior, Tired often and / or hard to awaken, Coma, etc.

 

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

•       Liver transplantation

 

The DelveInsight Urea Cycle Disorders market report gives a thorough understanding of the Urea Cycle Disorders by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis and treatment. 

 

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Urea Cycle Disorders Epidemiology  

 

Urea cycle disorders are autosomal recessive disorders with the exception of ornithine transcarbamylase deficiency, which is X-linked. Neonates with absent urea cycle enzyme activity typically present with hyperammonemic coma within the first week after birth. Outcomes can be severe, with high morbidity and mortality approaching 50% (Batshaw and Monahan, 1987). This disorder is one of the most common inborn errors of metabolism in the liver, with an estimated Incidence ranging from 1:30,000 to 1:46,000 live births. Here are some of the epidemiological studies.

 

The Urea Cycle Disorders epidemiology division provide insights about historical and current Urea Cycle Disorders patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.  

 

Urea Cycle Disorders Drug Chapters

 

The dynamics of 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 of 2020–2030.   

 

Drug chapter segment of the Urea Cycle Disorders report encloses the detailed analysis of Urea Cycle Disorders marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Urea Cycle Disorders clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

 

Marketed Drugs 

The report provides the details of the marketed product available for Urea Cycle Disorders treatment. 

 

Urea Cycle Disorders Emerging Drugs 

The report provides the details of the emerging therapies under the late and mid-stage of development for Urea Cycle Disorders treatment. 

 

Urea Cycle Disorders Market Outlook

Recent advances in the treatment of inborn errors of urea synthesis have significantly decreased mortality. The treatment of urea cycle disorders consists of dietary management to limit ammonia production, in conjunction with medications and/or supplements which provide alternative pathways for the removal of ammonia from the bloodstream. The aim is to correct biochemical abnormalities and ensure adequate nutritional intake. Treatment involves compounds that increase the removal of nitrogen waste. These compounds convert nitrogen into products other than urea, which are then excreted; hence, the load on the urea cycle is reduced. The first compounds to be used were sodium benzoate and arginine. Later, phenylacetate was used, which has now been replaced by phenylbutyrate.

 

Hemodialysis is very effective at reducing plasma ammonia and should immediately be initiated if elevated hyperammonemia is observed. Ammonia scavenger medications such as Ammonul IV are also useful. Ammonul IV acts by removing glycine and glutamate from plasma thereby their reducing contribution to ammonia formation.

 

The Urea Cycle Disorders market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Urea Cycle Disorders market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

 

This segment gives a thorough detail of Urea Cycle Disorders market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, 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. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight. 

 

According to DelveInsight, Urea Cycle Disorders market in 7MM is expected to change in the study period 2017-2030.

 

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