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Phenylketonuria - Epidemiology Forecast-2030

Phenylketonuria - Epidemiology Forecast-2030

  • February 2021
  • 100 pages
  • ID: 6028052
  • Format: PDF
  • Delve Insight

Summary

Table of Contents

‘Phenylketonuria—Epidemiology Forecast–2030’ report delivers an in-depth understanding of the PKU, historical and forecasted epidemiology as well as the PKU trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Phenylketonuria Disease Understanding
Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is characterized by the absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. Amino acids are the chemical building blocks of proteins and are essential for proper growth and development. With normal PAH activity, phenylalanine is converted to another amino acid, tyrosine. However, when PAH absent or deficient, phenylalanine accumulates and is toxic to the brain. Without treatment, most people with PKU would develop a severe intellectual disability. To prevent intellectual disability, treatment consists of a carefully controlled, phenylalanine-restricted diet beginning during the first days or weeks of life.
PKU is inherited in families in an autosomal recessive pattern. Autosomal recessive inheritance means that a person has two copies of the gene that is altered. Usually, each parent of an individual who has PKU carries one copy of the altered gene. Since each parent also has a normal gene, they do not show signs or symptoms of PKU.
Continued in the report…..
Phenylketonuria Epidemiology Perspective
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2018 to 2030.
Phenylketonuria Detailed Epidemiology Segmentation
• In 2020, the total diagnosed prevalent cases of Phenylketonuria were found out to be 49,887 in the 7MM, these cases are expected to grow at a CAGR of 0.58%, for the study period 2018–2020.
• The estimates suggest higher diagnosed prevalent cases of Phenylketonuria in the United States with 17,670 cases in 2020.
• The highest number of cases were in the age group <14 years that accounted for 69.41% of the PKU cases and the least cases were found in 45+ years which accounted for 4.03% of the total PKU cases in 2020, in the US.
• The total number of cases Classic PKU (cPKU) was the 10,355 followed by Mild PKU (mPKU) with 4,806 and Mild hyperphenylalaninemia (MHP) with 2,509 cases in 2020, in the US.
• Japan had 1,015 diagnosed prevalent cases of Phenylketonuria in 2020.
“With the increasing advancement and technologies, it has been observed that almost every newborn goes for the newborn screening process, adding up to 100% diagnosed rate; it can be concluded that almost all the cases of PKU are diagnosed in early age only. In very rare cases it is diagnosed in an older age group. Also, as per the KOLs views, most of the cases of PKU are diagnosed in the newborn screening process.”

Scope of the Report
• The report covers the descriptive overview of Phenylketonuria, explaining its causes, symptoms, pathophysiology, and genetic basis.
• The report provides insight into the 7MM historical and forecasted patient pool covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
• The report assesses the disease risk and burden and highlights the unmet needs of Phenylketonuria.
• The report helps to recognize the growth opportunities in the 7MM with respect to the patient population.
• The report provides the segmentation of the disease epidemiology for 7MM by Diagnosed Prevalent cases of Phenylketonuria (PKU), Age-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU), Severity-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU) and Diagnosed Prevalent Cases of Phenylketonuria (PKU) by Mutation type.

Report Highlights
• 10-Year Forecast of Phenylketonuria
• 7MM Coverage
• Diagnosed Prevalent cases of Phenylketonuria (PKU)
• Age-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU)
• Severity-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU)
• Diagnosed Prevalent Cases of Phenylketonuria (PKU) by Mutation type

Key Questions Answered
• What are the disease risk, and burden of Phenylketonuria?
• What is the historical Phenylketonuria patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
• What would be the forecasted patient pool of Phenylketonuria at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Phenylketonuria?
• Out of the above-mentioned countries, which country would have the highest prevalent population of Phenylketonuria during the forecast period (2021–2030)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?

Reasons to buy
The Phenylketonuria report will allow the user to -
• Develop business strategies by understanding the trends shaping and driving the 7MM Phenylketonuria epidemiology forecast.
• The Phenylketonuria epidemiology report and model were written and developed by Masters and Ph.D. level epidemiologists.
• The Phenylketonuria epidemiology model developed is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over the 10-year forecast period using reputable sources.

Key Assessments
• Patient Segmentation
• Disease Risk and Burden
• Risk of disease by the segmentation
• Factors driving growth in a specific patient population

Geographies Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2018–2030

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