Pompe Disease - Opportunity Assessment and Forecast to 2030

Table of Contents
1 Executive Summary
1.1 Executive Summary
2 Disease Overview
2.1 Definition of Pompe Disease
2.2 Pompe SWOT Analysis
2.3 Etiology and Pathophysiology
3 Epidemiology
3.1 Diagnosed Prevalent Cases of PD, 2020-2030
3.2 Diagnosed Prevalent Cases of PD by Specific Type, 2020
3.3 Sources and Methodology
3.4 Diagnosis of Pompe Disease - Treatable Population
4 Current Treatment Options
4.1 Treatment Paradigm
4.2 Marketed Products
4.3 Product Profile: Genzyme and Sanofi's Algucosidase alfa (Myozyme)
4.4 Product Profile: Genzyme and Sanofi's Algucosidase alfa (Lumizyme)
5 Unmet Needs and Opportunities
5.1 Unmet Needs in PD
5.2 Earlier Disease Diagnosis
5.3 Therapies that Restore GAA Expression in the CNS
5.4 Treatment with Improved Efficacy
5.5 Treatment with a Less Invasive Route of Administration
5.6 Reduced Cost annd Improved Criteria for Treatment Access
6 Pipeline Assessment
6.1 Pompe Disease: Pipeline Overview
6.2 Leading Pipeline Agents
6.3 Product Profile: avalglucosidase alfa
6.4 Product Profile: cipaglucosidase alfa
6.5 Product Profile: ACTUS-101
6.6 Product Profile: AVR-SPK-3006
6.7 Product Profile: AT-845
6.8 Pipeline Products - Review Designations
6.9 Pompe Disease: Clinical Trials Overview
7 R&D Strategies
7.1 Trends in Clinical Trial Design in Pompe Disease
7.2 Trends in Deal-Making in Pompe Disease
8 Market Outlook
8.1 Pompe Disease Market Growth
8.2 Pompe Disease Market Forecast
8.3 Market Drivers and Barriers
9 Appendix
9.1 Primary Research: KOL Information
9.2 About the Authors
10 Contact Us

List of Figures
Figure 1: Summary of Key Findings