Phenylketonuria Epidemiology Forecast Report 2021-2030 - ResearchAndMarkets.com

The "Phenylketonuria - Epidemiology Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

This '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.

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.

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.

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)?

Key Assessments

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

Key Topics Covered:

1 Key Insights

2 Report Introduction

3 Phenylketonuria (PKU) Epidemiology Overview at a Glance

3.1 Patient Distribution (%) of Phenylketonuria (PKU) in 2018

3.2 Patient Distribution (%) of Phenylketonuria (PKU) in 2030

4 Executive Summary of Phenylketonuria (PKU)

5 Disease Background and Overview

5.1 Introduction

5.2 Inheritance of PKU

5.3 Classification of PKU

5.4 Pathophysiology of PKU

5.5 Symptoms of PKU

5.6 Prognosis of PKU

5.7 Diagnosis of PKU

5.7.1 Bacterial Inhibition Test

5.7.2 Tandem Mass Spectrometry

5.7.3 Molecular Analysis of PKU-Associated PAH Mutations

5.7.4 Genotype-phenotype correlation

5.8 Differential Diagnosis

5.9 Diagnosis Guidelines

5.9.1 ACMG Practice Guidelines

5.1 Key European guidelines for the diagnosis and management of patients with PKU

6 Epidemiology and Patient Population

6.1 Key Findings

6.2 Methodology

6.3 Diagnosed Prevalent Cases of Phenylketonuria (PKU) in the 7MM

6.4 Age-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU) in the 7MM

6.5 Severity-specific Diagnosed Prevalent Cases of Phenylketonuria (PKU) in the 7MM

6.6 Diagnosed Prevalent Cases of Phenylketonuria (PKU) by Mutation type in the 7MM

6.7 The United States

6.5.1. Assumptions and Rationale

6.5.2. Diagnosed Prevalent Cases of Phenylketonuria in the United States

6.5.3. Age-specific Diagnosed Prevalent Cases of Phenylketonuria in the United States

6.5.4. Severity-specific Diagnosed Prevalent Cases of Phenylketonuria in the United States

6.5.5. Diagnosed Prevalence of Phenylketonuria by mutation type in the United States

6.8 EU5

6.8.1 Germany

6.8.2 France

6.8.3 Italy

6.8.4 Spain

6.8.5 United Kingdom

6.9 Japan

7 Patient Journey

8 KOL Views

9 Appendix

For more information about this report visit https://www.researchandmarkets.com/r/s7ool2

View source version on businesswire.com: https://www.businesswire.com/news/home/20210301005765/en/