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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

Published By :

GBI Research

Published Date : May 2012

Category :

Therapeutic Area

No. of Pages : 138 Pages


GBI Research, the leading business intelligence provider, has released its latest research, “Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity”, which provides insights into the global orphan diseases in genetic disorders therapeutics market, including market forecasts until 2018. The report provides an in-depth analysis of major orphan genetic disease indications, covering Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Fabry disease and Pompe disease. The report also includes insights into the orphan genetic disease therapeutics R&D pipeline. It analyzes the competitive landscape, including M&As, and licensing and co-development deals. The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research’s team of industry experts. 

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.
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  • Develop key strategic initiatives by understanding the key focus areas and top selling therapeutics of leading companies.
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Table of Contents

1 Table of Contents
1 Table of Contents
1.1 List of Tables
1.2 List of Figures

2 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Introduction

3 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Market Overview
3.1 Introduction
3.1.1 Orphan Disease Regulation and Incentives
3.2 Revenue Forecasts for Orphan Disease Therapeutics in Genetic Disorders
3.2.1 Revenue
3.2.2 Annual Cost of Treatment
3.3 Global Treatment Usage Patterns
3.3.1 Diseased Population
3.3.2 Treatment Seeking Population
3.3.3 Diagnosed Population
3.3.4 Prescription Population
3.4 Drivers and Restraints for Orphan Genetic Disease Therapeutics Market
3.4.1 Drivers for Orphan Genetic Disease Therapeutics Market
3.4.2 Restraints for Orphan Genetic Disease Therapeutics Market
3.4.3 Unmet Needs
3.4.4 Opportunities

4 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Geographical Landscape
4.1 The US
4.1.1 Revenue
4.1.2 Annual Cost of Treatment
4.1.3 Treatment Usage Patterns
4.2 Top Five Countries of Europe
4.2.1 Revenue
4.2.2 Annual Cost of Treatment
4.2.3 Treatment Usage Patterns
4.3 Japan
4.3.1 Revenue
4.3.2 Annual Cost of Treatment
4.3.3 Treatment Usage Patterns

5 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Therapeutic Landscape
5.1 Cystic Fibrosis
5.1.1 Introduction
5.1.2 Symptoms
5.1.3 Diagnosis
5.1.4 Treatment Flow
5.1.5 Revenue
5.1.6 Annual Cost of Treatment
5.1.7 Treatment Usage Patterns
5.1.8 Marketed Products
5.1.9 Drivers and Restraints for the Cystic Fibrosis Therapeutics Market
5.2 Duchenne Muscular Dystrophy
5.2.1 Introduction
5.2.2 Symptoms
5.2.3 Treatment Algorithm
5.2.4 Revenue
5.2.5 Annual Cost of Treatment
5.2.6 Treatment Usage Patterns
5.2.7 Marketed Therapies
5.2.8 Drivers and Restraints for Duchenne Muscular Dystrophy Therapeutics Market
5.3 Fabry Disease
5.3.1 Introduction
5.3.2 Symptoms
5.3.3 Treatment Flow
5.3.4 Revenue
5.3.5 Annual Cost of Treatment
5.3.6 Treatment Usage Patterns
5.3.7 Drivers and Restarints for Fabry Disease Therapeutics Market
5.3.8 Restraints for Fabry Disease Therapeutics Market
5.3.9 Marketed Products
5.4 Pompe Disease
5.4.1 Introduction
5.4.2 Symptoms
5.4.3 Treatment Flow Algorithm
5.4.4 Revenue
5.4.5 Annual Cost of Treatment
5.4.6 Treatment Usage Patterns
5.4.7 Marketed Products

6 Orphan Disease Therapeutics in Genetic Disorders to 2018 –Pipeline Analysis
6.1 Introduction
6.2 R&D Pipeline – Cystic Fibrosis
6.2.1 Pre-Clinical Stage
6.2.2 Phase I
6.2.3 Phase II
6.2.4 Phase III
6.2.5 Promising Drugs in the Pipeline
6.3 R&D Pipeline – Duchenne Muscular Dystrophy
6.3.1 Pre-Clinical
6.3.2 Phase I
6.3.3 Phase II
6.3.4 Phase III
6.3.5 Promising Molecules in the Pipeline
6.4 R&D Pipeline – Fabry Disease
6.4.1 Pre-Clinical
6.4.2 Phase II
6.4.3 Phase III
6.5 R&D Pipeline – Pompe Disease
6.5.1 Pre-Clinical
6.5.2 Phase II

7 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Competitive Landscape
7.1 Competitive Profiling
7.1.1 GlaxoSmithKline
7.1.2 Prosensa
7.1.3 Santhera
7.1.4 AVI BioPharma
7.1.5 Genzyme (Sanofi Subsidiary)
7.1.6 Shire
7.1.7 PTC Therapeutics
7.1.8 Novartis
7.1.9 Genentech (Roche)
7.1.10 Gilead

8 Orphan Disease Therapeutics in Genetic Disorders to 2018 – Strategic Consolidations
8.1 M&A Landscape
8.1.1 Key Deals
8.1.2 M&A Deals by Year
8.1.3 M&A Deals by Geography
8.1.4 M&A Deals by Value
8.2 R&D Licensing Agreements
8.2.1 Key Deals
8.2.2 Deals by Year
8.2.3 Deals by Geography
8.3 Co-Development Agreements
8.3.2 Deals by Year
8.3.3 Deals by Geography

9 Appendix
9.1 Market Definitions
9.2 Abbreviations
9.3 Sources
9.4 Research Methodology
9.4.1 Coverage
9.4.2 Secondary Research
9.4.3 Primary Research
9.4.4 Forecasting
9.4.5 Geographical Landscape
9.4.6 Pipeline Analysis
9.4.7 Competitive Landscape
9.4.8 Expert Panel Validation
9.5 Contact Us
9.6 Disclaimer

List of Table


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

List of Chart


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

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