Phenylketonuria (commonly known as PKU) is an inherited disorder that increases the levels of phenylalanine in the blood; phenylalanine is a building block of proteins (an amino acid). If the amino acid levels remain high for long, brain development slows down, which can subsequently cause mental retardation.
PKU symptoms can be mild or severe. These typically include a musty odor in the breath, skin or urine, caused by too much phenylalanine in the body. Neurological problems associated with the condition include seizures, hyperactivity, intellectual disability, delayed development, behavioral, emotional and social problems, and psychiatric disorders because phenylalanine can not transform into melanin.
Different forms of PKU with different clinical phenotypes have been described depending on the enzyme defect, the genotype, and the severity of the disease, establishing different classifications for PKU phenotypes. PKU may be classified as classic PKU and as variant PKU, which includes all milder forms of PKU (i.e., moderate PKU and mild PKU), as mild hyperphenylalaninemia (HPA) or non-PKU HPA, and, additionally, as BH4-responsive PKU.
The DelveInsight Phenylketonuria epidemiology report gives a thorough understanding of the Phenylketonuria by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis.
It also provides treatment algorithms and treatment guidelines for Phenylketonuria in the US, Europe, and Japan. The report covers the detailed information of the Phenylketonuria epidemiology scenario in seven major countries (US, EU5, and Japan).
Key Highlights Of The Report
According to Genetics Home Reference, the occurrence of PKU varies among ethnic groups and geographic regions worldwide. In the United States, PKU occurs in 1 in 10,000–15,000 newborns.
As per the study conducted by Wegberg et al. titled “The complete European guidelines on phenylketonuria: diagnosis and treatment,” the prevalence of PKU vary worldwide, and in Europe, the mean prevalence is approximately 1 in 10,000 newborns.
The study conducted by Trefz et al., reported prevalence of PKU among newborns to be 1 in 6,000 live births. Additionally, a period prevalence of 10.13 in 2015 (per 100,000 individuals) was found in adult patients. Most adult PKU patients were female (58.1%), and the mean age was found to be 50.9 ± 20.4 years.
According to DelveInsight’s analysis, Phenylketonuria (PKU) affects both the genders equally
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Delveinsight’s Phenylketonuria epidemiology division provides insights about historical and current patient pool and forecasted trends for every seven major countries.
The Phenylketonuria epidemiology data are studied through all possible divisions to give a better understanding of the Disease scenario in 7MM.
The Phenylketonuria epidemiology segment covers the epidemiology data in the US, EU5 countries (Germany, Spain, Italy, France, and the UK), and Japan from 2017 to 2030.
It also helps to recognize the causes of current and forecasted trends by exploring numerous studies, survey reports and views of key opinion leaders.
Scope of the Report
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The report provides insight into the historical and forecasted patient pool of Phenylketonuria in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan.
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The report assesses the disease risk and burden and highlights the unmet needs of Phenylketonuria.
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The report helps to recognize the growth opportunities in the 7MM with respect to the patient population.
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The Phenylketonuria report provides a detailed overview explaining Phenylketonuria causes, symptoms, classification, pathophysiology, diagnosis and treatment patterns.
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The Phenylketonuria Epidemiology Report and Model provide an overview of the risk factors and global trends of Phenylketonuria in the seven major markets (7MM:US, France, Germany, Italy, Spain, UK, and Japan).
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The report provides the segmentation of the Phenylketonuria epidemiology.
Key Questions Covered In The Phenylketonuria Report Include:
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What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Phenylketonuria?
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What are the key findings pertaining to the Phenylketonuria epidemiology across 7MM and which country will have the highest number of patients during the forecast period (2017-2030)?
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What would be the total number of patients of Phenylketonuria across the 7MM during the forecast period (2017-2030)?
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Among the EU5 countries, which country will have the highest number of patients during the forecast period (2017-2030)?
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At what CAGR the patient population is expected to grow in 7MM during the forecast period (2017-2030)?
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What is the disease risk, burden and unmet needs of the Phenylketonuria?
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What are the currently available treatments of Phenylketonuria?
Key Benefit of Phenylketonuria Epidemiology Report
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The Phenylketonuria Epidemiology report will allow the user to –
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Develop business strategies by understanding the trends shaping and driving the global Phenylketonuria market
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Quantify patient populations in the global Phenylketonuria market to improve product design, pricing, and launch plans
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Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Phenylketonuria therapeutics in each of the markets covered
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Understand the magnitude of Phenylketonuria population by its epidemiology
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The Phenylketonuria Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based with transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over 11-year forecast period using reputable sources
Table of Contents
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Key Insights
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Executive Summary of Phenylketonuria
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Phenylketonuria Disease Background and Overview
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Phenylketonuria Sign and Symptoms
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Phenylketonuria Pathophysiology
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Phenylketonuria Risk Factors
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Phenylketonuria Diagnosis
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Phenylketonuria Patient Journey
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Phenylketonuria Epidemiology and Patient Population
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Phenylketonuria Treatment Algorithm, Current Treatment, and Medical Practices
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KOL Views on Phenylketonuria
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Phenylketonuria Unmet Needs
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Appendix
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DelveInsight Capabilities
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Disclaimer
*The table of contents is not exhaustive; will be provided in the final report
About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.
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