Table of Contents PART 01: Executive summary • Highlights PART 02: Scope of the report • Market overview • Top-vendor offerings PART 03: Market research methodology • Research methodology • Economic indicators PART 04: Introduction • Key market highlights PART 05: Disease overview • Understanding the disease • Symptoms • Diagnosis • Etiology and pathogenesis • Biochemical pathway • Epidemiology • Management • Economic burden PART 06: Market landscape • Market overview • Five forces analysis PART 07: Market segmentation by type of Pompe disease PART 08: Geographical segmentation • Pompe disease treatment market in EMEA • Pompe disease treatment market in Americas • Pompe disease treatment market in US • Pompe disease treatment market in APAC PART 09: Market drivers • Special regulatory drug designations for orphan drugs • Reimbursement policies for treatment • Designated under ICD-10 code • Shorter development timelines PART 10: Impact of drivers PART 11: Market challenges • Limited patient population • Weak pipeline • Lack of long-term commitment PART 12: Impact of drivers and challenges PART 13: Market trends • Focus on gene therapy • Emergence of immunotherapy for Pompe disease • Growing public awareness PART 14: Vendor landscape • Competitive scenario • Other prominent vendors PART 15: Key vendor analysis • Amicus Therapeutics • Audentes Therapeutics • Sanofi Genzyme PART 16: Appendix • List of abbreviations PART 17: Explore Technavio List of Exhibits Exhibit 01: Product offerings Exhibit 02: Approved drugs for Pompe disease Exhibit 03: Snapshot of Pompe disease Exhibit 04: Signs and symptoms of Pompe disease Exhibit 05: Differential diagnosis of Pompe diseases in infants Exhibit 06: Differential diagnosis of Pompe diseases in adults and children Exhibit 07: Biochemical pathway of Pompe disease Exhibit 08: Approaches used in treating Pompe disease Exhibit 09: Snapshot of global Pompe disease treatment market 2015 Exhibit 10: Market overview of global Pompe disease treatment market Exhibit 11: Opportunity analysis in developed and emerging markets Exhibit 12: Global Pompe disease treatment market 2015-2020 ($ millions) Exhibit 13: Five forces analysis Exhibit 14: Market segmentation by type of Pompe disease Exhibit 15: Snapshot of global Pompe disease treatment market by geography Exhibit 16: Global Pompe disease treatment market segmentation by geography 2015-2020 ($ millions) Exhibit 17: Overview of Pompe disease treatment market in EMEA Exhibit 18: Prevalence rates of Pompe disease (per 100,000) in EMEA 2015 Exhibit 19: Incentives provided for orphan drugs by EU regulation Exhibit 20: Pompe disease treatment market in EMEA 2015-2020 ($ millions) Exhibit 21: Overview of Pompe disease treatment market in Americas Exhibit 22: Prevalence rates of Pompe disease (one in 100,000) in Americas 2015 Exhibit 23: Orphan drug development and regulatory challenges Exhibit 24: Pompe disease treatment market in Americas 2015-2020 ($ millions) Exhibit 25: Incentives provided for orphan drugs by US regulation Exhibit 26: Pompe disease treatment market in US 2015-2020 ($ millions) Exhibit 27: Overview of Pompe disease treatment market in APAC Exhibit 28: Prevalence rates of Pompe disease (one in 100,000) in APAC Exhibit 29: Incentives provided for orphan drugs by Japanese regulation Exhibit 30: Incentives provided for orphan drugs by Australian regulation Exhibit 31: Pompe disease treatment market in APAC 2015-2020 ($ millions) Exhibit 32: US FDA filed to approved time: Orphan drugs versus non-orphan drugs (median time) Exhibit 33: Impact of drivers Exhibit 34: Pipeline for Pompe disease treatment 2016 Exhibit 35: Pipeline molecules being developed for the treatment of Pompe disease Exhibit 36: Pompe disease drugs discontinued from development Exhibit 37: Impact of drivers and challenges Exhibit 38: Competitive structure analysis of global Pompe disease treatment market 2015 Exhibit 39: Region-wise combined sales of Myozyme and Lumizyme 2013-2015 ($ millions) Exhibit 40: Amicus Therapeutics: Key highlights Exhibit 41: Amicus Therapeutics: Strength assessment Exhibit 42: Amicus Therapeutics: Strategy assessment Exhibit 43: Amicus Therapeutics: Opportunity assessment Exhibit 44: Audentes Therapeutics: Profile Exhibit 45: Audentes Therapeutics: Strength assessment Exhibit 46: Audentes Therapeutics: Strategy assessment Exhibit 47: Audentes Therapeutics: Opportunity assessment Exhibit 48: Sanofi Genzyme: Key highlights Exhibit 49: Sanofi Genzyme: Strength assessment Exhibit 50: Sanofi Genzyme: Strategy assessment Exhibit 51: Sanofi Genzyme: Opportunity assessment

Amicus Therapeutics, Audentes Therapeutics, Sanofi-Genzyme, EpiVax, Oxyrane, Sangamo BioSciences, Valerion Therapeutics.