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1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3

2 Executive Summary 4
2.1 Highly Innovative and Diverse Pipeline 4
2.2 Alignment of Innovation to Genetics and Disease Processes 4
2.3 Deals Landscape Present Substantial Investment Opportunities 4

3 The Case for Innovation 5
3.1 Growing Opportunities for Biologic Products 6
3.2 Diversification of Molecular Targets 6
3.3 Innovative First-in-Class Product Developments Remain Attractive 6
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 7
3.5 Sustained Innovation 7
3.6 GBI Research Report Guidance 8

4 Clinical and Commercial Landscape 9
4.1 Disease Overview 9
4.2 Disease Epidemiology and Etiology 9
4.2.1 Disease Inheritance 10
4.3 Disease Pathophysiology 10
4.3.1 Hypothesized Pathophysiological Mechanisms of Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy 11
4.3.2 Dysfunction in Regeneration and Development of Fibrosis 13
4.4 Disease Symptoms 13
4.5 Complications and Co-morbidities 14
4.5.1 Respiratory Complications 14
4.5.2 Cardiac Complications 14
4.5.3 Scoliosis 14
4.6 Diagnosis 14
4.7 Disease Staging and Prognosis 15
4.8 Treatment Options 16
4.8.1 Pharmacological Treatments 16
4.8.2 Non-pharmacological Treatments 18
4.8.3 Treatment Algorithm 19
4.9 Overview of Marketed Products 21
4.9.1 Glucocorticoids 21
4.9.2 Translarna (ataluren) 23
4.9.3 Molecule Type and Target Analysis 23
4.10 Current Unmet Needs 24

5 Assessment of Pipeline Product Innovation 25
5.1 Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Pipeline by Molecule Type, Phase and Therapeutic Target 25
5.2 Comparative Distribution of Programs between Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Market, and Pipeline by Therapeutic Target Family 29

6 Signaling Pathways, Genetics and Innovation Alignment 34
6.1 First-in-Class Target Matrix Assessment 34
6.2 Repositioning Potential among Muscular Dystrophies 36

7 First-in-Class Target Evaluation 38
7.1 Pipeline Programs Targeting Sarcospan 38
7.2 Pipeline Programs Targeting Utrophin 40
7.3 Pipeline Programs Targeting Biglycan 43
7.4 Pipeline Programs Targeting Laminin-111 45
7.5 Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase 48
7.6 Pipeline Programs which Target G Protein-Coupled Receptor Mas 50
7.7 Pipeline Programs Targeting Mothers against Decapentaplegic Homolog 3 52
7.8 Pipeline Programs which Target Myostatin 54
7.9 Pipeline Programs Targeting Dystrophin 56
7.10 Conclusion 59

8 Deals and Strategic Consolidations 60
8.1 Industry-Wide First-in-Class Deals 60
8.2 Licensing Deals 61
8.3 Co-development Deals 66
8.4 First-in-Class Programs Not Involved in Licensing or Co-Development Deals 68

9 Appendix 70
9.1 Abbreviations 70
9.2 References 70
9.3 Contact Us 77
9.4 Disclaimer 77