Table of Contents
GBI Research Report Guidance 2
Executive Summary 3
Table of Contents 4

Introduction 8
Genetic Disorder Cluster Muscular Dystrophy Report Coverage 8
Becker Muscular Dystrophy – Overview 8
Duchenne Muscular Dystrophy – Overview 8
Therapeutics Development 9
Becker Muscular Dystrophy 9
Duchenne Muscular Dystrophy 12
Therapeutics Assessment 22
Becker Muscular Dystrophy 22
Duchenne Muscular Dystrophy 30
Companies Involved in Therapeutics Development 38
Becker Muscular Dystrophy 38
Duchenne Muscular Dystrophy 40
Dormant Projects 63
Becker Muscular Dystrophy 63
Duchenne Muscular Dystrophy 64
Discontinued Products 66
Duchenne Muscular Dystrophy 66
Product Development Milestones 67
Duchenne Muscular Dystrophy 67
Appendix 78
Methodology 78
Coverage 78
Secondary Research 78
Primary Research 78
Expert Panel Validation 78
Contact Us 79
Disclaimer 79

List of Tables
Number of Products under Development for Becker Muscular Dystrophy 9
Number of Products under Development by Companies, Becker Muscular Dystrophy 10
Products under Development by Companies, Becker Muscular Dystrophy 11
Number of Products under Development for Duchenne Muscular Dystrophy 12
Number of Products under Development by Companies, Duchenne Muscular Dystrophy 14
Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16
Products under Development by Companies, Duchenne Muscular Dystrophy 17
Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 21
Number of Products by Stage and Target, Becker Muscular Dystrophy 23
Number of Products by Stage and Mechanism of Action, Becker Muscular Dystrophy 25
Number of Products by Stage and Route of Administration, Becker Muscular Dystrophy 27
Number of Products by Stage and Molecule Type, Becker Muscular Dystrophy 29
Number of Products by Stage and Target, Duchenne Muscular Dystrophy 31
Number of Products by Stage and Mechanism of Action, Duchenne Muscular Dystrophy 33
Number of Products by Stage and Route of Administration, Duchenne Muscular Dystrophy 35
Number of Products by Stage and Molecule Type, Duchenne Muscular Dystrophy 37
Becker Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 38
Becker Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 38
Becker Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 39
Becker Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 39
Becker Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 40
Duchenne Muscular Dystrophy – Pipeline by Acceleron Pharma Inc 40
Duchenne Muscular Dystrophy – Pipeline by Akashi Therapeutics Inc 41
Duchenne Muscular Dystrophy – Pipeline by Asklepios BioPharmaceutical Inc 41
Duchenne Muscular Dystrophy – Pipeline by BioMarin Pharmaceutical Inc 41
Duchenne Muscular Dystrophy – Pipeline by Bristol-Myers Squibb Company 42
Duchenne Muscular Dystrophy – Pipeline by Capricor Therapeutics Inc 42
Duchenne Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 43
Duchenne Muscular Dystrophy – Pipeline by Catabasis Pharmaceuticals Inc 43
Duchenne Muscular Dystrophy – Pipeline by CRISPR Therapeutics 44
Duchenne Muscular Dystrophy – Pipeline by Daiichi Sankyo Company Ltd 44
Duchenne Muscular Dystrophy – Pipeline by Debiopharm International SA 45
Duchenne Muscular Dystrophy – Pipeline by Editas Medicine Inc 45
Duchenne Muscular Dystrophy – Pipeline by Eloxx Pharmaceuticals Ltd 46
Duchenne Muscular Dystrophy – Pipeline by FibroGen Inc 46
Duchenne Muscular Dystrophy – Pipeline by Galapagos NV 47
Duchenne Muscular Dystrophy – Pipeline by Genethon SA 47
Duchenne Muscular Dystrophy – Pipeline by GTx Inc 48
Duchenne Muscular Dystrophy – Pipeline by GW Pharmaceuticals Plc 48
Duchenne Muscular Dystrophy – Pipeline by Idera Pharmaceuticals Inc 49
Duchenne Muscular Dystrophy – Pipeline by Italfarmaco SpA 49
Duchenne Muscular Dystrophy – Pipeline by La Jolla Pharmaceutical Company 50
Duchenne Muscular Dystrophy – Pipeline by Lexicon Pharmaceuticals Inc 50
Duchenne Muscular Dystrophy – Pipeline by Marina Biotech Inc 51
Duchenne Muscular Dystrophy – Pipeline by Merck KGaA 51
Duchenne Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 52
Duchenne Muscular Dystrophy – Pipeline by Mitobridge Inc 52
Duchenne Muscular Dystrophy – Pipeline by Mitochon Pharmaceuticals Inc 53
Duchenne Muscular Dystrophy – Pipeline by MyoTherix Inc 53
Duchenne Muscular Dystrophy – Pipeline by NicOx SA 54
Duchenne Muscular Dystrophy – Pipeline by Nippon Shinyaku Co Ltd 54
Duchenne Muscular Dystrophy – Pipeline by Nobelpharma Co Ltd 55
Duchenne Muscular Dystrophy – Pipeline by Novartis AG 55
Duchenne Muscular Dystrophy – Pipeline by Pfizer Inc 56
Duchenne Muscular Dystrophy – Pipeline by Pluristem Therapeutics Inc 56
Duchenne Muscular Dystrophy – Pipeline by Prothelia Inc 57
Duchenne Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 57
Duchenne Muscular Dystrophy – Pipeline by RASRx LLC 58
Duchenne Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 58
Duchenne Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG 59
Duchenne Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 60
Duchenne Muscular Dystrophy – Pipeline by Strykagen Corp 61
Duchenne Muscular Dystrophy – Pipeline by Summit Therapeutics Plc 61
Duchenne Muscular Dystrophy – Pipeline by Taiho Pharmaceutical Co Ltd 62
Duchenne Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd 62
Becker Muscular Dystrophy – Dormant Projects 63
Duchenne Muscular Dystrophy – Dormant Projects 64
Duchenne Muscular Dystrophy – Discontinued Products 66

List of Figures
Number of Products under Development for Becker Muscular Dystrophy 9
Number of Products under Development by Companies, Becker Muscular Dystrophy 10
Number of Products under Development for Duchenne Muscular Dystrophy 12
Number of Products under Development by Companies, Duchenne Muscular Dystrophy 13
Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16
Number of Products by Targets, Becker Muscular Dystrophy 22
Number of Products by Stage and Targets, Becker Muscular Dystrophy 22
Number of Products by Mechanism of Actions, Becker Muscular Dystrophy 24
Number of Products by Stage and Mechanism of Actions, Becker Muscular Dystrophy 24
Number of Products by Routes of Administration, Becker Muscular Dystrophy 26
Number of Products by Stage and Routes of Administration, Becker Muscular Dystrophy 26
Number of Products by Molecule Types, Becker Muscular Dystrophy 28
Number of Products by Stage and Molecule Types, Becker Muscular Dystrophy 28
Number of Products by Top 10 Targets, Duchenne Muscular Dystrophy 30
Number of Products by Stage and Top 10 Targets, Duchenne Muscular Dystrophy 30
Number of Products by Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32
Number of Products by Stage and Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32
Number of Products by Routes of Administration, Duchenne Muscular Dystrophy 34
Number of Products by Stage and Routes of Administration, Duchenne Muscular Dystrophy 34
Number of Products by Molecule Types, Duchenne Muscular Dystrophy 36
Number of Products by Stage and Molecule Types, Duchenne Muscular Dystrophy 36