Table of Contents PART 01: Executive summary PART 02: Scope of the report PART 03: Research methodology PART 04: Introduction • Market outline PART 05: Understanding LSDs • Introduction PART 06: Pipeline PART 07: Market landscape • Market overview • Five forces analysis PART 08: Market segmentation by therapy • Enzyme replacement therapy (ERT) • Substrate reduction therapy (SRT) • Cystine depleting agents PART 09: Market segmentation by geography • Geographical segmentation • LSDs market in EMEA • LSDs market in Americas • LSDs market in APAC PART 10: Decision framework PART 11: Drivers and challenges • Market drivers • Market challenges PART 12: Trends • Advent of modern technologies • Regulatory assistance in emerging nations • Increased R&D activities • Increased focus on gene therapy • Emergence of immunotherapy • Growing public awareness PART 13: Vendor landscape • Competitive scenario PART 14: Key vendor analysis • Actelion Pharmaceuticals • BioMarin • Genzyme • Shire • Other Prominent Vendors PART 15: Appendix • List of abbreviations List of Exhibits  Exhibit 01: Classification of LSDs Exhibit 02: Pipeline snapshot Exhibit 03: Pipeline landscape based on various LSDs Exhibit 04: Key clinical trials Exhibit 05: Global LSDs market 2016-2021 ($ millions) Exhibit 06: Opportunity analysis of the global LSDs market Exhibit 07: Five forces analysis Exhibit 08: Global LSDs market segmentation by therapy Exhibit 09: Global LSDs market share by therapy 2016 Exhibit 10: Global LSDs ERT market 2016-2021 ($ millions) Exhibit 11: Potential benefits of using ERT for the treatment of various LSDs Exhibit 12: Global LSDs SRT market 2016-2021 ($ millions) Exhibit 13: Global LSDs cystine depleting agents market 2016-2021 ($ millions) Exhibit 14: Global LSDs market share by geography 2016 and 2021 Exhibit 15: Global LSDs market revenue by geography 2016-2021 Exhibit 16: Global LSDs market share by geography 2016-2021 Exhibit 17: Market scenario in EMEA Exhibit 18: LSDs market in EMEA 2016-2021 ($ millions) Exhibit 19: Incentives provided for orphan drugs by EU regulation Exhibit 20: Market scenario in Americas Exhibit 21: LSDs market in Americas 2016-2021 ($ millions) Exhibit 22: Orphan drug development and regulatory challenges Exhibit 23: Incentives provided for orphan drugs by US regulation Exhibit 24: LSDs market in APAC 2016-2021 ($ millions) Exhibit 25: Incentives provided for orphan drugs by Japanese regulation Exhibit 26: Incentives provided for orphan drugs by Australian regulation Exhibit 27: ICD-10 coverage for some of the LSDs Exhibit 28: US FDA filed to approved time: Orphan drugs versus non-orphan drugs and others (median time in months) Exhibit 29: Usefulness of DNA-based testing Exhibit 30: Competitive structure analysis of global LSDs market 2016 Exhibit 31: Actelion Pharmaceuticals: Key highlights Exhibit 32: Actelion Pharmaceuticals: Strength assessment Exhibit 33: Actelion Pharmaceuticals: Strategy assessment Exhibit 34: Actelion Pharmaceuticals: Opportunity assessment Exhibit 35: BioMarin: Key highlights Exhibit 36: BioMarin: Strength assessment Exhibit 37: BioMarin: Strategy assessment Exhibit 38: BioMarin Pharmaceuticals: Opportunity assessment Exhibit 39: Genzyme: Key highlights Exhibit 40: Genzyme: Strength assessment Exhibit 41: Genzyme: Strategy assessment Exhibit 42: Genzyme: Opportunity assessment Exhibit 43: Shire: Key highlights Exhibit 44: Shire: Strength assessment Exhibit 45: Shire: Strategy assessment Exhibit 46: Shire: Opportunity assessment

Actelion Pharmaceuticals, BioMarin, Genzyme, and Shire, Alexion Pharmaceuticals, Amicus Therapeutics, Chiesi Farmaceutici, Greenovation Biotech and FGK Clinical Research, Horizon Pharma, Leadiant Biosciences, Mylan, Pfizer, Protalix, Raptor, Recordati, Synageva BioPharma, Valerion Therapeutics, and Vtesse.