Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity

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Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity

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Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity

Summary

GBI Research found that the global orphan disease therapeutics in genetic disorders market was worth $1.5 billion in 2010, having grown from $559m in 2004 at a Compound Annual Growth Rate (CAGR) of 17.3%. It is expected to increase to $4.1 billion by 2018, growing at a CAGR of 13.8% over the period from 2010. This high rate of growth in the historic period can be attributed to the entry of novel, highly priced therapies into markets with high unmet need. These include Myozyme for Pompe disease and enzyme replacement therapies for Fabry disease, which have high costs of over $200,000 per patient per year. More highly-priced therapies are expected in the forecast period, to further drive growth. The recent approval of Kalydeco in CF and the expected approval of exon-skipping therapies for DMD are examples of this. Competitive activity is also increasing as larger companies realize the rewards of investing in orphan therapeutics, and the high unmet need and low competition in many markets is expected to attract more into the space in the future.

Scope

- Data and analysis on the global orphan disease therapeutics in genetic disorders in the leading geographies of the world– the US, the UK, Germany, France, Italy, Spain, and Japan.- Annualized market data for the orphan disease therapeutics in genetic disorders market from 2002–2010 with forecasts to 2018.- Market data on the therapeutic landscape, covering CF, DMD, Fabry disease and Pompe disease. This includes market size, market share, annual cost of therapy, treatment flow algorithm and branded and generic share.- Key drivers and restraints that have had a significant impact on the market and on each indication.- The competitive landscape of the global orphan disease therapeutics in genetic disorders, including companies such as Genzyme, GlaxoSmithKline, Novartis, Santhera, and Shire.- Key M&A activities and licensing and co-development agreements that took place from 2006–2011 in the global orphan disease therapeutics in genetic disorders market.

Reasons to buy

- Align your product portfolio to the markets with high growth potential.- Develop market-entry and market expansion strategies by identifying the potential region and global orphan disease therapeutics in genetic disorders market segments poised for strong growth.- Create a more tailored country strategy through the understanding of key drivers and barriers of the global oncology therapeutics market.- Develop key strategic initiatives by understanding the key focus areas and top selling therapeutics of leading companies.- Accelerate and strengthen your market position by identifying key companies for mergers, acquisitions and strategic partnerships.

Table of Contents

1 Table of Contents1 Table of Contents 51.1 List of Tables 81.2 List of Figures 112 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Introduction 133 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Market Overview 143.1 Introduction 143.1.1 Orphan Disease Regulation and Incentives 143.2 Revenue Forecasts for Orphan Disease Therapeutics in Genetic Disorders 163.2.1 Revenue 163.2.2 Annual Cost of Treatment 183.3 Global Treatment Usage Patterns 193.3.1 Diseased Population 193.3.2 Treatment Seeking Population 203.3.3 Diagnosed Population 213.3.4 Prescription Population 223.4 Drivers and Restraints for Orphan Genetic Disease Therapeutics Market 233.4.1 Drivers for Orphan Genetic Disease Therapeutics Market 233.4.2 Restraints for Orphan Genetic Disease Therapeutics Market 253.4.3 Unmet Needs 253.4.4 Opportunities 254 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Geographical Landscape 264.1 The US 264.1.1 Revenue 264.1.2 Annual Cost of Treatment 274.1.3 Treatment Usage Patterns 284.2 Top Five Countries of Europe 324.2.1 Revenue 324.2.2 Annual Cost of Treatment 334.2.3 Treatment Usage Patterns 344.3 Japan 384.3.1 Revenue 384.3.2 Annual Cost of Treatment 394.3.3 Treatment Usage Patterns 405 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Therapeutic Landscape 445.1 Cystic Fibrosis 445.1.1 Introduction 445.1.2 Symptoms 455.1.3 Diagnosis 455.1.4 Treatment Flow 465.1.5 Revenue 475.1.6 Annual Cost of Treatment 495.1.7 Treatment Usage Patterns 505.1.8 Marketed Products 545.1.9 Drivers and Restraints for the Cystic Fibrosis Therapeutics Market 585.2 Duchenne Muscular Dystrophy 615.2.1 Introduction 615.2.2 Symptoms 615.2.3 Treatment Algorithm 625.2.4 Revenue 635.2.5 Annual Cost of Treatment 665.2.6 Treatment Usage Patterns 675.2.7 Marketed Therapies 715.2.8 Drivers and Restraints for Duchenne Muscular Dystrophy Therapeutics Market 715.3 Fabry Disease 735.3.1 Introduction 735.3.2 Symptoms 735.3.3 Treatment Flow 745.3.4 Revenue 745.3.5 Annual Cost of Treatment 765.3.6 Treatment Usage Patterns 785.3.7 Drivers and Restarints for Fabry Disease Therapeutics Market 825.3.8 Restraints for Fabry Disease Therapeutics Market 835.3.9 Marketed Products 835.4 Pompe Disease 855.4.1 Introduction 855.4.2 Symptoms 865.4.3 Treatment Flow Algorithm 865.4.4 Revenue 875.4.5 Annual Cost of Treatment 885.4.6 Treatment Usage Patterns 895.4.7 Marketed Products 946 Orphan Disease Therapeutics in Genetic Disorders to 2018 –Pipeline Analysis 966.1 Introduction 966.2 R&D Pipeline – Cystic Fibrosis 976.2.1 Pre-Clinical Stage 976.2.2 Phase I 986.2.3 Phase II 996.2.4 Phase III 1026.2.5 Promising Drugs in the Pipeline 1026.3 R&D Pipeline – Duchenne Muscular Dystrophy 1046.3.1 Pre-Clinical 1046.3.2 Phase I 1056.3.3 Phase II 1066.3.4 Phase III 1066.3.5 Promising Molecules in the Pipeline 1076.4 R&D Pipeline – Fabry Disease 1086.4.1 Pre-Clinical 1086.4.2 Phase II 1096.4.3 Phase III 1096.5 R&D Pipeline – Pompe Disease 1106.5.1 Pre-Clinical 1106.5.2 Phase II 1107 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Competitive Landscape 1117.1 Competitive Profiling 1117.1.1 GlaxoSmithKline 1117.1.2 Prosensa 1127.1.3 Santhera 1127.1.4 AVI BioPharma 1137.1.5 Genzyme (Sanofi Subsidiary) 1147.1.6 Shire 1147.1.7 PTC Therapeutics 1157.1.8 Novartis 1167.1.9 Genentech (Roche) 1177.1.10 Gilead 1178 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Strategic Consolidations 1198.1 M&A Landscape 1198.1.1 Key Deals 1198.1.2 M&A Deals by Year 1218.1.3 M&A Deals by Geography 1218.1.4 M&A Deals by Value 1228.2 R&D Licensing Agreements 1228.2.1 Key Deals 1228.2.2 Deals by Year 1268.2.3 Deals by Geography 1278.3 Co-Development Agreements 1278.3.2 Deals by Year 1328.3.3 Deals by Geography 1329 Appendix 1339.1 Market Definitions 1339.2 Abbreviations 1339.3 Sources 1349.4 Research Methodology 1359.4.1 Coverage 1359.4.2 Secondary Research 1359.4.3 Primary Research 1369.4.4 Forecasting 1369.4.5 Geographical Landscape 1379.4.6 Pipeline Analysis 1379.4.7 Competitive Landscape 1379.4.8 Expert Panel Validation 1389.5 Contact Us 1389.6 Disclaimer 138

List of Tables

1.1 List of TablesTable 1: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue ($m), 2004–2010 16Table 2: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts ($m), 2010–2018 16Table 3: Orphan Disease Therapeutics in Genetic Disorders, Global, Average ACT ($), 2004–2010 18Table 4: Orphan Disease Therapeutics in Genetic Disorders, Global, Average ACT ($), 2010–2018 18Table 5: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2004–2010 19Table 6: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2010–2018 19Table 7: Orphan Disease Therapeutics in Genetic Disorders, Global, Treatment Seeking Population, 2004–2010 20Table 8: Orphan Disease Therapeutics in Genetic Disorders, Global, Treatment Seeking Population, 2010–2018 20Table 9: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2004–2010 21Table 10: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2010–2018 21Table 11: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2004–2010 22Table 12: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2010–2018 22Table 13: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue ($m), 2004–2010 26Table 14: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue Forecasts ($m), 2010–2018 26Table 15: Orphan Disease Therapeutics in Genetic Disorders, The US, Average ACT ($), 2004–2010 27Table 16: Orphan Disease Therapeutics in Genetic Disorders, The US, Average ACT ($), 2010–2018 27Table 17: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2004–2010 28Table 18: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2010–2018 28Table 19: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2004–2010 29Table 20: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2010–2018 29Table 21: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2004–2010 30Table 22: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2010–2018 30Table 23: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2004–2010 31Table 24: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2010–2018 31Table 25: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue ($m), 2004–2010 32Table 26: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue Forecasts ($m), 2010–2018 32Table 27: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Average ACT ($), 2004–2010 33Table 28: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Average ACT ($), 2010–2018 33Table 29: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe Diseased Population, 2004–2010 34Table 30: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diseased Population, 2010–2018 34Table 31: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2004–2010 35Table 32: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2010–2018 35Table 33: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2004–2010 36Table 34: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2010–2018 36Table 35: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2004–2010 37Table 36: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2010–2018 37Table 37: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue ($m), 2004–2010 38Table 38: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue Forecasts ($m), 2010–2018 38Table 39: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2004–2010 39Table 40: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2010–2018 39Table 41: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2004–2010 40Table 42: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2010–2018 40Table 43: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2004–2010 41Table 44: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2010–2018 41Table 45: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2004–2010 42Table 46: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2010–2018 42Table 47: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2004–2010 43Table 48: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2010–2018 43Table 49: Cystic Fibrosis Therapeutics Market, Global, Revenue ($m), 2004–2010 47Table 50: Cystic Fibrosis Therapeutics Market, Global, Revenue Forecasts ($m), 2010–2018 47Table 51: Cystic Fibrosis Therapeutics Market, Global, Average ACT ($), 2004–2010 49Table 52: Cystic Fibrosis Therapeutics Market, Global, Average ACT ($), 2010–2018 49Table 53: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2004–2010 50Table 54: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2010–2018 50Table 55: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population , 2004–2010 51Table 56: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population, 2010–2018 51Table 57: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2004–2010 52Table 58: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2010–2018 52Table 59: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2004–2010 53Table 60: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2010–2018 53Table 61: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenue ($m), 2004–2010 63Table 62: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenues Forecasts ($m), 2010–2018 63Table 63: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2004–2010 66Table 64: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2010–2018 66Table 65: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2004–2010 67Table 66: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2010–2018 67Table 67: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2004–2010 68Table 68: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2010–2018 68Table 69: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diagnosed Population, 2004–2010 69Table 70: Duchenne Muscular Dystrophy Therapeutics Market, Japan, Diagnosed Population, 2010–2018 69Table 71: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2004–2010 70Table 72: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2010–2018 70Table 73: Fabry Disease Therapeutics Market, Global, Revenue ($m), 2004–2010 74Table 74: Fabry Disease Therapeutics Market, Global, Revenue Forecasts ($m), 2010–2018 74Table 75: Fabry Disease Therapeutics Market, Global, Average ACT ($), 2004–2010 76Table 76: Fabry Disease Therapeutics Market, Global, Average ACT ($), 2010–2018 76Table 77: Fabry Disease Therapeutics Market, Global, Diseased Population, 2004–2010 78Table 78: Fabry Disease Therapeutics Market, Global, Diseased Population, 2010–2018 78Table 79: Fabry Disease Therapeutics Market, Global, Treatment Seeking Population, 2004–2010 79Table 80: Fabry Disease Therapeutics Market, Global,Treatment Seeking Population, 2010–2018 79Table 81: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2004–2010 80Table 82: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2010–2018 80Table 83: Fabry Disease Therapeutics Market, Global, Prescription Population, 2004–2010 81Table 84: Fabry Disease Therapeutics Market, Global, Prescription Population, 2010–2018 81Table 85: Pompe Disease Therapeutics Market, Global, Revenue ($m), 2004–2010 87Table 86: Pompe Disease Therapeutics Market, Global, Revenues Forecasts ($m), 2010–2018 87Table 87: Pompe Disease Therapeutics Market, Global, Average ACT ($), 2004–2010 88Table 88: Pompe Disease Therapeutics Market, Global, Average ACT ($), 2010–2018 88Table 89: Pompe Disease Therapeutics Market, Global, Diseased Population, 2004–2010 89Table 90: Pompe Disease Therapeutics Market, Global, Diseased Population, 2010–2018 89Table 91: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2004–2010 90Table 92: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2010–2018 90Table 93: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2004–2010 91Table 94: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2010–2018 91Table 95: Pompe Disease Therapeutics Market, Global, Prescription Population, 2004–2010 92Table 96: Pompe Disease Therapeutics Market, Global, Prescription Population, 2010–2018 92Table 97: Cystic Fibrosis Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 97Table 98: Cystic Fibrosis Therapeutics Market, Global, Phase I Pipeline, 2011 98Table 99: Cystic Fibrosis Therapeutics Market, Global, Phase II Pipeline, 2011 99Table 100: Cystic Fibrosis Therapeutics Market, Global, Phase III Pipeline, 2011 102Table 101: Duchenne Muscular Dystrophy Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 104Table 102: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase I Pipeline, 2011 105Table 103: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase II Pipeline, 2011 106Table 104: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase II Pipeline, 2011 106Table 105: Fabry Disease Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 108Table 106: Fabry Disease Therapeutics Market, Global, Phase II Pipeline, 2011 109Table 107: Fabry Disease Therapeutics Market, Global, Phase III Pipeline, 2011 109Table 108: Pompe Disease Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 110Table 109: Pompe Disease Therapeutics Market, Global, Phase II Pipeline, 2011 110

List of Figures

1.2 List of FiguresFigure 1: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts ($m), 2004–2018 16Figure 2: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts by Region ($m), 2004–2018 17Figure 3: Orphan Disease Therapeutics in Genetic Disorders, Global, ACT ($), 2004–2018 18Figure 4: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2004–2018 19Figure 5: Orphan Disease Therapeutics i`n Genetic Disorders Market, Global, Treatment Seeking Population, 2004–2018 20Figure 6: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2004–2018 21Figure 7: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2004–2018 22Figure 8: Orphan Disease Therapeutics in Genetic Disorders, Global, Drivers and Restraints 23Figure 9: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue ($m), 2004–2018 26Figure 10: Orphan Disease Therapeutics in Genetic Disorders, The US, ACT ($), 2004–2018 27Figure 11: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2004–2018 28Figure 12: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2004–2018 29Figure 13: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2004–2018 30Figure 14: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2004–2018 31Figure 15: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue ($m), 2004–2018 32Figure 16: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, ACT ($), 2004–2018 33Figure 17: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diseased Population, 2004–2018 34Figure 18: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2004–2018 35Figure 19: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2004–2018 36Figure 20: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2004–2018 37Figure 21: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue ($m), 2004–2018 38Figure 22: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2004–2018 39Figure 23: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2004–2018 40Figure 24: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2004–2018 41Figure 25: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2004–2018 42Figure 26: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2004–2018 43Figure 27: Cystic Fibrosis Therapeutics Market, Global, Treatment Flow Algorithm, 2011 46Figure 28: Cystic Fibrosis Therapeutics Market, Global, Revenue ($m), 2004–2018 47Figure 29: Cystic Fibrosis Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2018 48Figure 30: Cystic Fibrosis Therapeutics Market, Global, ACT ($), 2004–2018 49Figure 31: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2004–2018 50Figure 32: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population, 2004–2018 51Figure 33: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2004–2018 52Figure 34: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2004–2018 53Figure 35: Cystic Fibrosis Therapeutics Market, Global, Drivers and Restraints 58Figure 36: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Flow Algorithm 62Figure 37: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenues ($m), 2004–2018 63Figure 38: Duchenne Muscular Dystrophy Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2018 64Figure 39: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2004–2018 66Figure 40: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2004–2018 67Figure 41: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2004–2018 68Figure 42: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diagnosed Population, 2004–2018 69Figure 43: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2004–2018 70Figure 44: Duchenne Muscular Dystrophy Therapeutics Market, Global, Drivers and Restraints 71Figure 45: Fabry Disease Therapeutics Market, Global, Treatment Flow Algorithm, 2011 74Figure 46: Fabry Disease Therapeutics Market, Global, Revenue ($m), 2004–2018 74Figure 47: Fabry Disease Therapeutics Market, Global, ACT ($), 2004–2018 76Figure 48: Fabry Disease Therapeutics Market, Global, Diseased Population, 2004–2018 78Figure 49: Fabry Disease Therapeutics Market, Global, Treatment Seeking Population, 2004–2018 79Figure 50: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2004–2018 80Figure 51: Fabry Disease Therapeutics Market, Global, Prescription Population, 2004–2018 81Figure 52: Fabry Disease Therapeutics Market, Global, Drivers and Restraints 82Figure 53: Pompe Disease Therapeutics Market, Global, Treatment Flow Algorithm 86Figure 54: Pompe Disease Therapeutics Market, Global, Revenue ($m), 2004–2018 87Figure 55: Pompe Disease Therapeutics Market, Global, ACT ($), 2004–2018 88Figure 56: Pompe Disease Therapeutics Market, Global, Diseased Population, 2004–2018 89Figure 57: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2004–2018 90Figure 58: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2004–2018 91Figure 59: Pompe Disease Therapeutics Market, Global, Prescription Population, 2004–2018 92Figure 60: Pompe Disease Therapeutics Market, Global, Drivers and Restraints 93Figure 61: Orphan Disease Therapeutics in Genetic Disorders, GSK, SWOT, 2011 111Figure 62: Orphan Disease Therapeutics in Genetic Disorders, Prosensa, SWOT, 2011 112Figure 63: Orphan Disease Therapeutics in Genetic Disorders, Santhera, SWOT, 2011 113Figure 64: Orphan Disease Therapeutics in Genetic Disorders, Avi BioPharma, SWOT, 2011 113Figure 65: Orphan Disease Therapeutics in Genetic Disorders, Genzyme (Sanofi Subsidiary), SWOT, 2011 114Figure 66: Orphan Disease Therapeutics in Genetic Disorders, Shire, SWOT, 2011 115Figure 67: Orphan Disease Therapeutics in Genetic Disorders, PTC Therapeutics, SWOT, 2011 115Figure 68: Orphan Disease Therapeutics in Genetic Disorders, Novartis, SWOT, 2011 116Figure 69: Orphan Disease Therapeutics in Genetic Disorders, Genentech (Roche), SWOT, 2011 117Figure 70: Orphan Disease Therapeutics in Genetic Disorders, Gilead, SWOT, 2011 118Figure 71: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Year, 2006–2011 121Figure 72: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Geography (%), 2006–2011 121Figure 73: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Value (%), 2006–2011 122Figure 74: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Licensing Deals by Year, 2007–2011 126Figure 75: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Licensing Deals by Geography (%), 2006–2011 127Figure 76: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Co-Development Deals by Year, 2006–2011 132Figure 77: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Co-Development Deals by Geography (%), 2006–2011 132