PART 01: EXECUTIVE SUMMARY PART 02: SCOPE OF THE REPORT • 2.1 Preface • 2.2 Preface • 2.3 Currency conversion rates for US$ PART 03: MARKET LANDSCAPE • Market ecosystem • Market characteristics • Market segmentation analysis PART 04: MARKET SIZING • Market definition • Market sizing 2018 • Market size and forecast 2018-2023 PART 05: FIVE FORCES ANALYSIS • Bargaining power of buyers • Bargaining power of suppliers • The threat of new entrants • Threat of substitutes • Threat of rivalry • Market condition PART 06: PIPELINE PART 07: MARKET SEGMENTATION BY TYPE • Market segmentation by type • Comparison by type • Biologics - Market size and forecast 2018-2023 • Small molecules - Market size and forecast 2018-2023 • Market opportunity by type PART 08: CUSTOMER LANDSCAPE PART 09: GEOGRAPHIC LANDSCAPE • Geographic segmentation • Geographic comparison • North America - Market size and forecast 2018-2023 • Europe - Market size and forecast 2018-2023 • Asia - Market size and forecast 2018-2023 • ROW - Market size and forecast 2018-202 • Key leading countries • Market opportunity PART 10: DECISION FRAMEWORK PART 11: DRIVERS AND CHALLENGES • Market drivers • Market challenges PART 12: MARKET TRENDS • Strong pipeline • Increasing research funding • Special drug designations PART 13: VENDOR LANDSCAPE • Overview • Landscape disruption • Competitive scenario PART 14: VENDOR ANALYSIS • Vendors covered • Vendor classification • Market positioning of vendors • F. Hoffmann-La Roche Ltd. • PTC Therapeutics Inc. • Santhera Pharmaceuticals • Sarepta Therapeutics Inc. • Wave Life Sciences Ltd. PART 15: APPENDIX • Research methodology • List of abbreviations • Definition of market positioning of vendors PART 16: EXPLORE TECHNAVIO   Exhibit 01: Product overview Exhibit 02: Global pharmaceuticals market Exhibit 03: Segments of global pharmaceuticals market Exhibit 04: Market characteristics Exhibit 05: Market segments Exhibit 06: Market definition - Inclusions and exclusions checklist Exhibit 07: Market size 2018 Exhibit 08: Global market: Size and forecast 2018-2023 ($ millions) Exhibit 09: Global market: Year-over-year growth 2019-2023 (%) Exhibit 10: Five forces analysis 2018 Exhibit 11: Five forces analysis 2023 Exhibit 12: Bargaining power of buyers Exhibit 13: Bargaining power of suppliers Exhibit 14: Threat of new entrants Exhibit 15: Threat of substitutes Exhibit 16: Threat of rivalry Exhibit 17: Market condition - Five forces 2018 Exhibit 18: Global DMD therapeutics market pipeline: Overview Exhibit 19: Global DMD therapeutics market pipeline: Snapshot Exhibit 20: Type - Market share 2018-2023 (%) Exhibit 21: Comparison by type Exhibit 22: Biologics - Market size and forecast 2018-2023 ($ millions) Exhibit 23: Biologics - Year-over-year growth 2019-2023 (%) Exhibit 24: Small molecules - Market size and forecast 2018-2023 ($ millions) Exhibit 25: Small molecules - Year-over-year growth 2019-2023 (%) Exhibit 26: Market opportunity by type Exhibit 27: Customer landscape Exhibit 28: Market share by geography 2018-2023 (%) Exhibit 29: Geographic comparison Exhibit 30: North America - Market size and forecast 2018-2023 ($ millions) Exhibit 31: North America - Year-over-year growth 2019-2023 (%) Exhibit 32: Top 3 countries in North America Exhibit 33: Europe - Market size and forecast 2018-2023 ($ millions) Exhibit 34: Europe - Year-over-year growth 2019-2023 (%) Exhibit 35: Top 3 countries in Europe Exhibit 36: Asia - Market size and forecast 2018-2023 ($ millions) Exhibit 37: Asia - Year-over-year growth 2019-2023 (%) Exhibit 38: Top 3 countries in Asia Exhibit 39: ROW - Market size and forecast 2018-2023 ($ millions) Exhibit 40: ROW - Year-over-year growth 2019-2023 (%) Exhibit 41: Top 3 countries in ROW Exhibit 42: Key leading countries Exhibit 43: Market opportunity Exhibit 44: Impact of drivers and challenges Exhibit 45: Vendor landscape Exhibit 46: Landscape disruption Exhibit 47: Vendors covered Exhibit 48: Vendor classification Exhibit 49: Market positioning of vendors Exhibit 50: F. Hoffmann-La Roche Ltd. - Vendor overview Exhibit 51: F. Hoffmann-La Roche Ltd. - Business segments Exhibit 52: F. Hoffmann-La Roche Ltd. - Organizational developments Exhibit 53: F. Hoffmann-La Roche Ltd. - Geographic focus Exhibit 54: F. Hoffmann-La Roche Ltd. - Segment focus Exhibit 55: F. Hoffmann-La Roche Ltd. - Key offerings Exhibit 56: F. Hoffmann-La Roche Ltd. - Key customers Exhibit 57: PTC Therapeutics Inc. - Vendor overview Exhibit 58: PTC Therapeutics Inc. - Business segments Exhibit 59: PTC Therapeutics Inc. - Organizational developments Exhibit 60: PTC Therapeutics Inc. - Key offerings Exhibit 61: PTC Therapeutics Inc. - Key customers Exhibit 62: Santhera Pharmaceuticals - Vendor overview Exhibit 63: Santhera Pharmaceuticals - Business segments Exhibit 64: Santhera Pharmaceuticals - Organizational developments Exhibit 65: Santhera Pharmaceuticals - Geographic focus Exhibit 66: Santhera Pharmaceuticals - Key offerings Exhibit 67: Santhera Pharmaceuticals - Key customers Exhibit 68: Sarepta Therapeutics Inc. - Vendor overview Exhibit 69: Sarepta Therapeutics Inc. - Business segments Exhibit 70: Sarepta Therapeutics Inc. - Organizational developments Exhibit 71: Sarepta Therapeutics Inc. - Geographic focus Exhibit 72: Sarepta Therapeutics Inc. - Key offerings Exhibit 73: Sarepta Therapeutics Inc. - Key customers Exhibit 74: Wave Life Sciences Ltd. - Vendor overview Exhibit 75: Wave Life Sciences Ltd. - Business segments Exhibit 76: Wave Life Sciences Ltd. - Organizational developments Exhibit 77: Wave Life Sciences Ltd. - Key offerings Exhibit 78: Wave Life Sciences Ltd. - Key customers Exhibit 79: Validation techniques employed for market sizing Exhibit 80: Definition of market positioning of vendors

F. Hoffmann-La Roche Ltd., PTC Therapeutics Inc., Santhera Pharmaceuticals, Sarepta Therapeutics Inc., and Wave Life Sciences Ltd.