Summary
Pompe disease is a rare multisystem disorder caused by pathogenic variations in the GAA gene, which contains the information for production and function of a protein called acid alpha-glucosidase (GAA). Because of the shortage of this protein (an enzyme), a complex sugar named glycogen cannot be degraded to a simple sugar like glucose. This causes the glycogen to accumulate in all kinds of tissues, but primarily in skeletal muscle, smooth muscle, and cardiac muscle, where it causes damage to tissue structure and function. Pompe disease is inherited as an autosomal recessive genetic trait.
For the 8MM, GlobalData epidemiologists provide two different forecasts for the diagnosed incident and diagnosed prevalent cases of Pompe disease-a forecast based on international registry data and a forecast adjusted for the underestimation of international registry-based data.
Based on international registry data, GlobalData epidemiologists estimated that the 8MM had 135 diagnosed incident cases of Pompe disease in 2017. GlobalData epidemiologists believe the registry underestimates the true incidence of Pompe disease. GlobalData estimates that there are 365 diagnosed incident cases of Pompe disease in the 8MM in 2017 after adjusting for the underestimation of international registry data. This is expected to increase to 373 incident cases by 2027.
GlobalData epidemiologists used the available data on Pompe disease to the best extent possible to provide the most insightful epidemiological forecast for the diagnosed incident cases and diagnosed prevalent cases of Pompe disease in the 8MM. GlobalData epidemiologists conducted an exhaustive literature review in order to find the best available data, and used the most appropriate incidence and prevalence estimates to the extent that they were available.
Scope
- The Pompe Disease Epidemiology Report provides an overview of the risk factors and global trends of Pompe Disease in the eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and Brazil). In addition, this report also includes a 10-year epidemiological forecast for the following segmentations for all ages across the 8MM.
- Diagnosed incident cases of Pompe disease.
- Diagnosed prevalent cases of Pompe disease.
- Diagnosed incident cases of Pompe disease by type.
- Diagnosed incident cases of late-onset Pompe disease (LOPD) by severity.
- Diagnosed incident cases of infantile-onset Pompe disease (IOPD) by cross-reactive immunological material (CRIM) status
- The Pompe Disease epidemiology report is written and developed by Masters- and PhD-level epidemiologists.
- The Epidemiology Report is in-depth, high quality, transparent and market-driven, providing expert analysis of disease trends in the 8MM.
Reasons to buy
The Pompe Disease Epidemiology report will allow you to -
- Develop business strategies by understanding the trends shaping and driving the global Pompe Disease market.
- Quantify patient populations in the global Pompe Disease market to improve product design, pricing, and launch plans.
- Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Pompe Disease therapeutics in each of the markets covered.
- Understand magnitude of Pompe Disease population by cross-reative immunological material status.
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1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 4
1.2 List of Figures 4
2 Pompe Disease: Executive Summary 5
2.1 Related Reports 8
2.2 Upcoming Reports 8
3 Epidemiology 9
3.1 Disease Background 9
3.2 Risk Factors and Comorbidities 10
3.3 Global and Historical Trends 10
3.4 Forecast Methodology 11
3.4.1 Sources 12
3.4.2 Forecast Assumptions and Methods 15
3.4.3 Diagnosed Incident Cases of Pompe Disease - Based on International Registry Data 15
3.4.4 Diagnosed Incident Cases of Pompe Disease by Type - Based on International Registry Data 16
3.4.5 Diagnosed Incident Cases of LOPD by Severity - Based on International Registry Data 17
3.4.6 Diagnosed Incident Cases of IOPD by CRIM Status - Based on International Registry Data 17
3.4.7 Diagnosed Prevalent Cases of Pompe Disease - Based on International Registry Data 18
3.4.8 Diagnosed Prevalent Cases of Pompe Disease - Method-1 18
3.4.9 Diagnosed Prevalent Cases of Pompe Disease - Method-2 19
3.4.10 Forecast Adjusted for the Underestimation of International Registry Data 19
3.5 Epidemiological Forecast for Pompe Disease (2017-2027) - Based on International Registry Data 20
3.5.1 Diagnosed Incident Cases of Pompe Disease 20
3.5.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease 21
3.5.3 Diagnosed Incident Cases of Pompe Disease by Type 22
3.5.4 Diagnosed Incident Cases of LOPD by Severity 23
3.5.5 Diagnosed Incident Cases of IOPD by CRIM Status 24
3.5.6 Diagnosed Prevalent Cases of Pompe Disease - Method-1 25
3.5.7 Diagnosed Prevalent Cases of Pompe Disease - Method-2 26
3.6 Epidemiological Forecast for Pompe Disease (2017-2027) - Adjusted for the Underestimation of International Registry Data 27
3.6.1 Diagnosed Incident Cases of Pompe Disease 27
3.6.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease 28
3.6.3 Diagnosed Incident Cases of Pompe Disease by Type 29
3.6.4 Diagnosed Incident Cases of LOPD by Severity 30
3.6.5 Diagnosed Incident Cases of IOPD by CRIM Status 31
3.6.6 Diagnosed Prevalent Cases of Pompe Disease - Method-1 32
3.6.7 Diagnosed Prevalent Cases of Pompe Disease - Method-2 33
3.7 Discussion 34
3.7.1 Epidemiological Forecast Insight 34
3.7.2 Limitations of the Analysis 35
3.7.3 Strengths of the Analysis 35
4 Appendix 36
4.1 Bibliography 36
4.2 About the Authors 38
4.2.1 Epidemiologist 38
4.2.2 Reviewers 38
4.2.3 Global Director of Therapy Analysis and Epidemiology 39
4.2.4 Global Head and EVP of Healthcare Operations and Strategy 39
4.3 About GlobalData 40
4.4 Contact Us 40
4.5 Disclaimer 40
1.1 List of Tables
Table 1: Risk Factors for Pompe Disease 10
1.2 List of Figures
Figure 1: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 and 2027 (Forecast Adjusted for the Underestimation of International Registry Data) 6
Figure 2: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-1, N, Both Sexes, All Ages, 2017 and 2027 (Forecast Adjusted for the Underestimation of International Registry Data) 7
Figure 3: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-2, N, Both Sexes, All Ages, 2017 and 2027 (Forecast Adjusted for the Underestimation of International Registry Data)) 8
Figure 4: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease 12
Figure 5: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Pompe Disease 13
Figure 6: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease by Type 13
Figure 7: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of LOPD by Severity 14
Figure 8: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of IOPD by CRIM Status 14
Figure 9: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 20
Figure 10: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Based on International Registry Data) 21
Figure 11: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 22
Figure 12: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 23
Figure 13: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 24
Figure 14: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-1, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 25
Figure 15: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-2, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data) 26
Figure 16: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 27
Figure 17: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 28
Figure 18: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 29
Figure 19: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 30
Figure 20: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 31
Figure 21: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-1, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 32
Figure 22: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-2, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data) 33
