Ornithine Transcarbamylase (OTC) Deficiency – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033


 Ornithine transcarbamylase (OTC) deficiency is an X-linked recessive disorder within the group of urea cycle disorders, leading to hyperammonemia. This disorder results from a deficiency in the enzyme responsible for catalyzing the formation of citrulline through the condensation of carbamyl phosphate and ornithine. In the absence of functional ornithine transcarbamylase, carbamyl phosphate accumulates in the cytoplasm, becoming available for de novo pyrimidine biosynthesis, consequently elevating levels of orotic and uracil in urine. Initial symptoms often include vomiting, increasing lethargy, irritability, and refusing to eat. Males tend to exhibit more severe symptoms and have a less favorable prognosis. The diagnosis of OTC deficiency relies on clinical manifestations, with plasma ammonia levels usually elevated (>200 µmol/L) when encephalopathy is present. Plasma amino acid analysis typically reveals decreased citrulline and arginine levels and increased glutamine. Urine organic acid analysis often shows elevated levels of orotic acid. Other potential diagnoses to consider are carbamoyl-phosphate synthetase deficiency, argininosuccinic aciduria, hyperammonemia due to N-acetylglutamate synthase deficiency, citrullinemia type 1, and argininemia. Prognosis varies depending on disease severity but is generally poor in patients with early neonatal disease. Timely diagnosis and treatment of hyperammonemic episodes are crucial for preventing neurological complications. Patients in a hyperammonemic coma require immediate treatment at a specialized tertiary care center. This treatment involves lowering plasma ammonia levels through methods such as hemodialysis or hemofiltration, implementing ammonia scavenger therapy, reversing catabolism using glucose and lipid infusions, and closely monitoring for neurological damage, including electroencephalogram surveillance and seizure treatment if necessary. Long-term therapy requires lifelong restriction of protein intake and nitrogen scavenger therapy, which may involve sodium benzoate and sodium or glycerol phenylbutyrate. In severe cases, particularly neonatal-onset OTC deficiency or frequent hyperammonemic episodes, liver transplantation may be considered, typically performed between 3 to 6 months of age.

·       The incidence of ornithine transcarbamylase is estimated to be 1/17000 in the USA.

 

Thelansis’s “Ornithine Transcarbamylase (OTC) Deficiency Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Ornithine Transcarbamylase (OTC) Deficiency treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of Ornithine Transcarbamylase (OTC) Deficiency across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Ornithine Transcarbamylase (OTC) Deficiency Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

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