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EpiCast Report: Duchenne Muscular Dystrophy - Epidemiology Forecast to 2023

Published By :

GlobalData

Published Date : Mar 2015

Category :

Diseases & Conditions

No. of Pages : 58 Pages


Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are rare progressive muscle wasting disorders.

GlobalData epidemiologists forecast that the diagnosed prevalent cases of DMD (all ages) in the 6MM will increase from 17,675 diagnosed prevalent cases in 2013 to 17,712 diagnosed prevalent cases in 2023, at an Annual Growth Rate (AGR) of 0.02%. The 6MM had an estimated 6,076 diagnosed prevalent cases of DMD for ages 5-13 years in 2013, and the number of diagnosed prevalent cases is expected to increase to 6,250 by 2023, at an AGR of 0.29%. GlobalData epidemiologists forecast that the diagnosed prevalent cases of DMD with nonsense mutations (all ages) in the 6MM will increase from 2,260 diagnosed prevalent cases in 2013 to 2,264 diagnosed prevalent cases in 2023, at an AGR of 0.02%.

GlobalData epidemiologists used the best available data from peer-reviewed journal articles and national DMD mutation databases to arrive at a meaningful and in-depth analysis of the diagnosed prevalent cases of DMD and BMD for men in the 6MM based on: a confirmed deletion, duplication, or point mutation in the DMD gene; reduced or absent dystrophin immunolabeling; or cases fulfilling “definite” or “probable” criteria of the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet). For all the 6MM, the diagnosed prevalent cases of DMD and BMD were segmented by age, which facilitates an understanding of the distribution of the disease within the population, and informs strategies to improve the management of the disease. The diagnosed prevalent cases of DMD in each of the 6MM were further segmented by therapeutically significant populations including the applicability of exon skipping therapies (exons 51, 45, 53, and 44), nonsense mutations, and cases ages 5-13 years in these markets.

Scope

  • The Duchenne Muscular Dystrophy (DMD) EpiCast Report provides an overview of the risk factors, comorbidities, and the global and historical trends for DMD in the six major markets (6MM) (US, France, Germany, Italy, Spain, and UK). It includes a 10-year epidemiological forecast for the diagnosed prevalent cases of DMD and Becker Muscular Dystrophy (BMD) in men, segmented by age (in 10-year age groups beginning at 0 years and ending at =40 years for cases of DMD with a special focus on the subgroup of ages 5-13 years, and in 15-year age groups beginning at 0 years and ending at =75 years for cases of BMD) in these markets. Additionally, the diagnosed prevalent cases of DMD (all ages) are further segmented by the applicability of exon skipping therapies (exons 51, 45, 53, and 44) and nonsense mutations.
  • The DMD epidemiology report is written and developed by Mastersand PhD-level epidemiologists.
  • The EpiCast Report is in-depth, high quality, transparent and market-driven, providing expert analysis of disease trends in the 6MM.

Reasons to buy

  • Develop business strategies by understanding the trends shaping and driving the global DMD market.
  • Quantify patient populations in the global DMD market to improve product design, pricing, and launch plans.
  • Organize sales and marketing efforts by identifying the sex and age groups that present the best opportunities for DMD therapeutics in each of the markets covered.
Table of Contents

1 Table of Contents
1 Table of Contents 6
1.1 List of Tables 8
1.2 List of Figures 9

2 Introduction 10
2.1 Catalyst 10
2.2 Related Reports 11
2.3 Upcoming Related Reports 11

3 Epidemiology 12
3.1 Disease Background 12
3.2 Risk Factors and Comorbidities 13
3.3 Global Trends 14
3.4 Forecast Methodology 16
3.4.1 Sources Used 21
3.4.2 Sources Not Used 23
3.4.3 Forecast Assumptions and Methods 24
3.5 Epidemiological Forecast for DMD (2013-2023) 29
3.5.1 Diagnosed Prevalent Cases of DMD (All Ages) 29
3.5.2 Diagnosed Prevalent Cases of DMD (Ages 5-13 Years) 31
3.5.3 Age-Specific Diagnosed Prevalent Cases of DMD (All Ages) 32
3.5.4 Age-Standardized Diagnosed Prevalence of DMD 34
3.6 Epidemiological Forecast for Nonsense Mutations in DMD (2013-2023) 35
3.7 Epidemiological Forecast for the Applicability of Exon Skipping Therapies in DMD (2013-2023) 37
3.7.1 Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy (All Ages) 37
3.7.2 Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy (All Ages) 38
3.7.3 Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy (All Ages) 40
3.7.4 Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy (All Ages) 41
3.8 Epidemiological Forecast for BMD (2013-2023) 43
3.8.1 Diagnosed Prevalent Cases of BMD (All Ages) 43
3.8.2 Age-Specific Diagnosed Prevalent Cases of BMD (All Ages) 44
3.8.3 Age-Standardized Diagnosed Prevalence of BMD 46
3.9 Discussion 47
3.9.1 Epidemiological Forecast Insight 47
3.9.2 Limitations of the Analysis 48
3.9.3 Strengths of the Analysis 49

4 Appendix 50
4.1 Bibliography 50
4.2 About the Authors 53
4.2.1 Epidemiologists 53
4.2.2 Reviewers 53
4.2.3 Global Director of Therapy Analysis and Epidemiology 54
4.2.4 Global Head of Healthcare 55
4.3 About GlobalData 56
4.4 About EpiCast 56
4.5 Disclaimer 57

List of Table


Table 1: Risk Factors and Comorbidities for DMD and BMD 14
Table 2: Global Estimates for the Diagnosed Prevalence of DMD and BMD 16
Table 3: MD STARnet Case Definition Criteria for DMD and BMD 18
Table 4: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD in the 6MM 19
Table 5: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of BMD in the 6MM 19
Table 6: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD with Nonsense Mutations in the 6MM 20
Table 7: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD Eligible for Exon Skipping Therapies (Exons 51, 45, 53, or 44) in the 6MM 21
Table 8: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, Select Years 2013-2023 30
Table 9: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, Select Years 2013-2023 31
Table 10: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N (Row %), 2013 33
Table 11: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, Select Years 2013-2023 36
Table 12: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 37
Table 13: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023 39
Table 14: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 40
Table 15: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 42
Table 16: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, Select Years 2013-2023 43
Table 17: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N (Row %), 2013 45

List of Chart


Figure 1: Patient Flow for the Epidemiological Forecast of DMD in the 6MM 17
Figure 2: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, 2013-2023 30
Figure 3: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, 2013-2023 32
Figure 4: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N, 2013 34
Figure 5: 6MM, Age-Standardized Diagnosed Prevalence of DMD (Cases per 100,000 Population), All Ages, Men, 2013 35
Figure 6: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, 2013-2023 36
Figure 7: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, 2013-2023 38
Figure 8: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023 39
Figure 9: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, 2013-2023 41
Figure 10: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, 2013-2023 42
Figure 11: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, 2013-2023 44
Figure 12: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N, 2013 46
Figure 13: 6MM, Age-Standardized Diagnosed Prevalence of BMD (Cases per 100,000 Population), All Ages, Men, 2013 47

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