1. Key Insights 2. Pompe Disease Market Overview at a Glance 2.1. Market Share (%) Distribution of Pompe Disease in 2017 2.2. Market Share (%) Distribution of Pompe Disease in 2028 3. Disease Background and Overview 3.1. Introduction 3.2. Types 3.3. Genetic defects 3.4. Clinical Manifestations 3.5. Causes 3.6. Signs and Symptoms 3.7. Pathogenesis 3.8. Diagnosis 4. Epidemiology and Patient Population – By Region 4.1. Key Findings 4.2. KOL Views 5. Ten Emerging Markets (EM) Total Prevalent Patient Population of Pompe Disease 5.1. Total Prevalence of Pompe Disease in 10 Emerging Markets 5.2. Diagnosed Prevalence of Pompe Disease in 10 Emerging Markets 6. Region Wise-Epidemiology of Pompe Disease 6.1. Asia (China and Taiwan) 6.1.1. Assumptions and Rationale 6.2. China 6.2.1. Total Live Birth Cases of Pompe Disease in China 6.2.2. Birth Prevalence of Pompe Disease by Onset Types in China 6.2.3. Total Prevalent Cases of Pompe Disease in China 6.2.4. Diagnosed Prevalence of Pompe Disease in China 6.3. Taiwan 6.3.1. Total Live Birth Cases of Pompe Disease in Taiwan 6.3.2. Birth Prevalence of Pompe Disease by Onset Types in Taiwan 6.3.3. Total Prevalent Cases of Pompe Disease in Taiwan 6.3.4. Diagnosed Prevalence of Pompe Disease in Taiwan 6.4. Middle East (Saudi Arabia and U.A.E.) 6.4.1. Assumptions and Rationale 6.5. Saudi Arabia 6.5.1. Total Live Birth Cases of Pompe Disease in Saudi Arabia 6.5.2. Birth Prevalence of Pompe Disease by Onset Types in Saudi Arabia 6.5.3. Total Prevalent Cases of Pompe Disease in Saudi Arabia 6.5.4. Diagnosed Prevalence of Pompe Disease in Saudi Arabia 6.6. U.A.E. 6.6.1. Total Live Birth Cases of Pompe Disease in U.A.E. 6.6.2. Birth Prevalence of Pompe Disease by Onset Types in U.A.E. 6.6.3. Total Prevalent Cases of Pompe Disease in U.A.E. 6.6.4. Diagnosed Prevalence of Pompe Disease in U.A.E. 6.7. Eastern Europe (Russia and Turkey) 6.7.1. Assumptions and Rationale 6.8. Russia 6.8.1. Total Live Birth Cases of Pompe Disease in Russia 6.8.2. Birth Prevalence of Pompe Disease by Onset Types in Russia 6.8.3. Total Prevalent Cases of Pompe Disease in Russia 6.8.4. Diagnosed Prevalence of Pompe Disease in Russia 6.9. Turkey 6.9.1. Total Live Birth Cases of Pompe Disease in Turkey 6.9.2. Birth Prevalence of Pompe Disease by Onset Types in Turkey 6.9.3. Total Prevalent Cases of Pompe Disease in Turkey 6.9.4. Diagnosed Prevalence of Pompe Disease in Turkey 6.10. LATAM (Mexico, Brazil, Argentina and Colombia) 6.10.1. Assumptions and Rationale 6.11. Mexico 6.11.1. Total Live Birth Cases of Pompe Disease in Mexico 6.11.2. Birth Prevalence of Pompe Disease by Onset Types in Mexico 6.11.3. Total Prevalent Cases of Pompe Disease in Mexico 6.11.4. Diagnosed Prevalence of Pompe Disease in Mexico 6.12. Brazil 6.12.1. Total Live Birth Cases of Pompe Disease in Brazil 6.12.2. Birth Prevalence of Pompe Disease by Onset Types in Brazil 6.12.3. Total Prevalent Cases of Pompe Disease in Brazil 6.12.4. Diagnosed Prevalence of Pompe Disease in Brazil 6.13. Argentina 6.13.1. Total Live Birth Cases of Pompe Disease in Argentina 6.13.2. Birth Prevalence of Pompe Disease by Onset Types in Argentina 6.13.3. Total Prevalent Cases of Pompe Disease in Argentina 6.13.4. Diagnosed Prevalence of Pompe Disease in Argentina 6.14. Colombia 6.14.1. Total Live Birth Cases of Pompe Disease in Colombia 6.14.2. Birth Prevalence of Pompe Disease by Onset Types in Colombia 6.14.3. Total Prevalent Cases of Pompe Disease in Colombia 6.14.4. Diagnosed Prevalence of Pompe Disease in Colombia 7. Treatment and Management of Pompe Disease 8. Unmet Needs 9. Marketed Drugs 9.1. Lumizyme: Genzyme 9.1.1. Product Description 9.1.2. Regulatory Milestones 9.1.3. Safety and Efficacy 9.1.4. Sales 9.1.5. Product Profile 10. Emerging Therapies 10.1. AT-GAA: Amicus Therapeutics 10.1.1. Product Description 10.1.2. Other Developmental Activities 10.1.3. Regulatory Milestones 10.1.4. Clinical Development 10.1.5. Safety and Efficacy 10.1.6. Product Profile 11. Other Promising Candidates 11.1. ACTUS-101: Actus Therapeutics 11.1.1. Product Description 11.1.2. Other Developmental Activities 11.1.3. Regulatory Milestones 11.1.4. Clinical Development 11.1.5. Product Profile 12. Pompe Disease: 10 EM Analysis 12.1. Key Findings 12.2. KOL Views 12.3. Market Size of Pompe Disease in 10 EM 12.3.1. Market Size of Pompe Disease by Therapies 13. Market Outlook by Country 13.1. Emerging Markets: Market Outlook 13.2. China 13.2.1. Total Market Size of Pompe Disease 13.2.2. Market Size of Pompe Disease by Therapies 13.3. Taiwan 13.3.1. Total Market Size of Pompe Disease 13.3.2. Market Size of Pompe Disease by Therapies 13.4. Saudi Arabia 13.4.1. Total Market Size of Pompe Disease 13.4.2. Market Size of Pompe Disease by Therapies 13.5. U.A.E. 13.5.1. Total Market Size of Pompe Disease 13.5.2. Market Size of Pompe Disease by Therapies 13.6. Russia 13.6.1. Total Market Size of Pompe Disease 13.6.2. Market Size of Pompe Disease by Therapies 13.7. Turkey 13.7.1. Total Market Size of Pompe Disease 13.7.2. Market Size of Pompe Disease by Therapies 13.8. Mexico 13.8.1. Total Market Size of Pompe Disease 13.8.2. Market Size of Pompe Disease by Therapies 13.9. Brazil 13.9.1. Total Market Size of Pompe Disease 13.9.2. Market Size of Pompe Disease by Therapies 13.10. Argentina 13.10.1. Total Market Size of Pompe Disease 13.10.2. Market Size of Pompe Disease by Therapies 13.11. Colombia 13.11.1. Total Market Size of Pompe Disease 13.11.2. Market Size of Pompe Disease by Therapies 14. Market Drivers 15. Market Barriers 16. Appendix 16.1. Report Methodology 17. DelveInsight Capabilities 18. Disclaimer 19. About DelveInsight

Table 1: Total Prevalence of Pompe Disease in 10 Emerging Markets (2017-2028) Table 2: Diagnosed Prevalence of Pompe Disease in 10 Emerging Markets (2017-2028) Table 3: Total Live Birth Cases of Pompe Disease in China (2017-2028) Table 4: Birth Prevalence of Pompe Disease by Onset Types in China (2017-2028) Table 5: Total Prevalent Cases of Pompe Disease in China (2017-2028) Table 6: Diagnosed Prevalence of Pompe Disease in China (2017-2028) Table 7: Total Live Birth Cases of Pompe Disease in Taiwan (2017-2028) Table 8: Birth Prevalence of Pompe Disease by Onset Types in Taiwan (2017-2028) Table 9: Total Prevalent Cases of Pompe Disease in Taiwan (2017-2028) Table 10: Diagnosed Prevalence of Pompe Disease in Taiwan (2017-2028) Table 11: Total Live Birth Cases of Pompe Disease in Saudi Arabia (2017-2028) Table 12: Birth Prevalence of Pompe Disease by Onset Types in Saudi Arabia (2017-2028) Table 13: Total Prevalent Cases of Pompe Disease in Saudi Arabia (2017-2028) Table 14: Diagnosed Prevalence of Pompe Disease in Saudi Arabia (2017-2028) Table 15: Total Live Birth Cases of Pompe Disease in U.A.E. (2017-2028) Table 16: Birth Prevalence of Pompe Disease by Onset Types in U.A.E. (2017-2028) Table 17: Total Prevalent Cases of Pompe Disease in U.A.E. (2017-2028) Table 18: Diagnosed Prevalence of Pompe Disease in U.A.E. (2017-2028) Table 19: Total Live Birth Cases of Pompe Disease in Russia (2017-2028) Table 20: Birth Prevalence of Pompe Disease by Onset Types in Russia (2017-2028) Table 21: Total Prevalent Cases of Pompe Disease in Russia (2017-2028) Table 22: Diagnosed Prevalence of Pompe Disease in Russia (2017-2028) Table 23: Total Live Birth Cases of Pompe Disease in Turkey (2017-2028) Table 24: Birth Prevalence of Pompe Disease by Onset Types in Turkey (2017-2028) Table 25: Total Prevalent Cases of Pompe Disease in Turkey (2017-2028) Table 26: Diagnosed Prevalence of Pompe Disease in Turkey (2017-2028) Table 27: Total Live Birth Cases of Pompe Disease in Mexico (2017-2028) Table 28: Birth Prevalence of Pompe Disease by Onset Types in Mexico (2017-2028) Table 29: Total Prevalent Cases of Pompe Disease in Mexico (2017-2028) Table 30: Diagnosed Prevalence of Pompe Disease in Mexico (2017-2028) Table 31: Total Live Birth Cases of Pompe Disease in Brazil (2017-2028) Table 32: Birth Prevalence of Pompe Disease by Onset Types in Brazil (2017-2028) Table 33: Total Prevalent Cases of Pompe Disease in Brazil (2017-2028) Table 34: Diagnosed Prevalence of Pompe Disease in Brazil (2017-2028) Table 35: Total Live Birth Cases of Pompe Disease in Argentina (2017-2028) Table 36: Birth Prevalence of Pompe Disease by Onset Types in Argentina (2017-2028) Table 37: Total Prevalent Cases of Pompe Disease in Argentina (2017-2028) Table 38: Diagnosed Prevalence of Pompe Disease in Argentina (2017-2028) Table 39: Total Live Birth Cases of Pompe Disease in Colombia (2017-2028) Table 40: Birth Prevalence of Pompe Disease by Onset Types in Colombia (2017-2028) Table 41: Total Prevalent Cases of Pompe Disease in Colombia (2017-2028) Table 42: Diagnosed Prevalence of Pompe Disease in Colombia (2017-2028) Table 43: AT-GAA, Clinical Trial Description, 2019 Table 44: ACTUS-101, Clinical Trial Description, 2019 Table 45: Total Market Size of Pompe Disease in 10 Emerging Markets in USD Million (2017-2028) Table 46: Total Market Size of Pompe Disease by Therapies in 10 Emerging Markets in USD Million (2017-2028) Table 47: Total Market Size of Pompe Disease in China, USD Million (2017-2028) Table 48: China Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 49: Market Size of Pompe Disease in Taiwan, USD Million (2017-2028) Table 50: Taiwan Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 51: Market Size of Pompe Disease in the Saudi Arabia, USD Million (2017-2028) Table 52: Saudi Arabia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 53: Market Size of Pompe Disease in U.A.E., USD Million (2017-2028) Table 54: U.A.E. Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 55: Market Size of Pompe Disease in Russia, USD Million (2017-2028) Table 56: Russia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 57: Market Size of Pompe Disease in Turkey, USD Million (2017-2028) Table 58: Turkey Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 59: Market Size of Pompe Disease in Mexico, USD Million (2017-2028) Table 60: Mexico Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 61: Market Size of Pompe Disease in Brazil, USD Million (2017-2028) Table 62: Brazil Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 63: Market Size of Pompe Disease in Argentina, USD Million (2017-2028) Table 64: Argentina Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Table 65: Market Size of Pompe Disease in Colombia, USD Million (2017-2028) Table 66: Colombia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 1: Comparison of normal physiology to pathophysiology in Pompe disease Figure 2: Pathophysiology of late-onset Pompe disease Figure 3: Diagnostic algorithm for infantile-onset Pompe disease. Figure 4: Diagnostic algorithm for late-onset (> 1 year) Pompe disease. Figure 5: Total Prevalence of Pompe Disease in 10 Emerging Markets (2017-2028) Figure 6: Diagnosed Prevalence of Pompe Disease in 10 Emerging Markets (2017-2028) Figure 7: Total Live Birth Cases of Pompe Disease in China (2017-2028) Figure 8: Birth Prevalence of Pompe Disease by Onset Types in China (2017-2028) Figure 9: Total Prevalent Cases of Pompe Disease in China (2017-2028) Figure 10: Diagnosed Prevalence of Pompe Disease in China (2017-2028) Figure 11: Total Live Birth Cases of Pompe Disease in Taiwan (2017-2028) Figure 12: Birth Prevalence of Pompe Disease by Onset Types in Taiwan (2017-2028) Figure 13: Total Prevalent Cases of Pompe Disease in Taiwan (2017-2028) Figure 14: Diagnosed Prevalence of Pompe Disease in Taiwan (2017-2028) Figure 15: Total Live Birth Cases of Pompe Disease in Saudi Arabia (2017-2028) Figure 16: Birth Prevalence of Pompe Disease by Onset Types in Saudi Arabia (2017-2028) Figure 17: Total Prevalent Cases of Pompe Disease in Saudi Arabia (2017-2028) Figure 18: Diagnosed Prevalence of Pompe Disease in Saudi Arabia (2017-2028) Figure 19: Total Live Birth Cases of Pompe Disease in U.A.E. (2017-2028) Figure 20: Birth Prevalence of Pompe Disease by Onset Types in U.A.E. (2017-2028) Figure 21: Total Prevalent Cases of Pompe Disease in U.A.E. (2017-2028) Figure 22: Diagnosed Prevalence of Pompe Disease in U.A.E. (2017-2028) Figure 23: Total Live Birth Cases of Pompe Disease in Russia (2017-2028) Figure 24: Birth Prevalence of Pompe Disease by Onset Types in Russia (2017-2028) Figure 25: Total Prevalent Cases of Pompe Disease in Russia (2017-2028) Figure 26: Diagnosed Prevalence of Pompe Disease in Russia (2017-2028) Figure 27: Total Live Birth Cases of Pompe Disease in Turkey (2017-2028) Figure 28: Birth Prevalence of Pompe Disease by Onset Types in Turkey (2017-2028) Figure 29: Total Prevalent Cases of Pompe Disease in Turkey (2017-2028) Figure 30: Diagnosed Prevalence of Pompe Disease in Turkey (2017-2028) Figure 31: Total Live Birth Cases of Pompe Disease in Mexico (2017-2028) Figure 32: Birth Prevalence of Pompe Disease by Onset Types in Mexico (2017-2028) Figure 33: Total Prevalent Cases of Pompe Disease in Mexico (2017-2028) Figure 34: Diagnosed Prevalence of Pompe Disease in Mexico (2017-2028) Figure 35: Total Live Birth Cases of Pompe Disease in Brazil (2017-2028) Figure 36: Birth Prevalence of Pompe Disease by Onset Types in Brazil (2017-2028) Figure 37: Total Prevalent Cases of Pompe Disease in Brazil (2017-2028) Figure 38: Diagnosed Prevalence of Pompe Disease in Brazil (2017-2028) Figure 39: Total Live Birth Cases of Pompe Disease in Argentina (2017-2028) Figure 40: Birth Prevalence of Pompe Disease by Onset Types in Argentina (2017-2028) Figure 41: Total Prevalent Cases of Pompe Disease in Argentina (2017-2028) Figure 42: Diagnosed Prevalence of Pompe Disease in Argentina (2017-2028) Figure 43: Total Live Birth Cases of Pompe Disease in Colombia (2017-2028) Figure 44: Birth Prevalence of Pompe Disease by Onset Types in Colombia (2017-2028) Figure 45: Total Prevalent Cases of Pompe Disease in Colombia (2017-2028) Figure 46: Diagnosed Prevalence of Pompe Disease in Colombia (2017-2028) Figure 47: Unmet Needs of Pompe Disease Figure 48: Total Market Size of Pompe Disease in 10 Emerging Markets in USD Million (2017-2028) Figure 49: Total Market Size of Pompe Disease by Therapies in 10 Emerging Markets in USD Million (2017-2028) Figure 50: Total Market Size of Pompe Disease in China, USD Million (2017-2028) Figure 51: China Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 52: Market Size of Pompe Disease in Taiwan, USD Million (2017-2028) Figure 53: Taiwan Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 54: Market Size of Pompe Disease in the Saudi Arabia, USD Million (2017-2028) Figure 55: Saudi Arabia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 56: Market Size of Pompe Disease in U.A.E., USD Million (2017-2028) Figure 57: U.A.E. Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 58: Market Size of Pompe Disease in Russia, USD Million (2017-2028) Figure 59: Russia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 60: Market Size of Pompe Disease in Turkey, USD Million (2017-2028) Figure 61: Turkey Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 62: Market Size of Pompe Disease in Mexico, USD Million (2017-2028) Figure 63: Mexico Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 64: Market Size of Pompe Disease in Brazil, USD Million (2017-2028) Figure 65: Brazil Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 66: Market Size of Pompe Disease in Argentina, USD Million (2017-2028) Figure 67: Argentina Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 68: Market Size of Pompe Disease in Colombia, USD Million (2017-2028) Figure 69: Colombia Market Size of Pompe Disease by Therapies in USD Million (2017-2028) Figure 70: Market Drivers Figure 71: Market Barriers

1. Genzyme 2. Amicus Therapeutics 3. Actus Therapeutics and Others