Table of Contents Table of Contents 2 List of Tables 6 List of Figures 8 Introduction 9 Global Markets Direct Report Coverage 9 Duchenne Muscular Dystrophy Overview 10 Therapeutics Development 11 Pipeline Products for Duchenne Muscular Dystrophy - Overview 11 Pipeline Products for Duchenne Muscular Dystrophy - Comparative Analysis 12 Duchenne Muscular Dystrophy - Therapeutics under Development by Companies 13 Duchenne Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 18 Duchenne Muscular Dystrophy - Pipeline Products Glance 19 Late Stage Products 19 Clinical Stage Products 20 Early Stage Products 21 Unknown Stage Products 22 Duchenne Muscular Dystrophy - Products under Development by Companies 23 Duchenne Muscular Dystrophy - Products under Investigation by Universities/Institutes 29 Duchenne Muscular Dystrophy - Companies Involved in Therapeutics Development 30 Acceleron Pharma, Inc. 30 Anagenesis Biotechnology 31 Asklepios BioPharmaceutical, Inc. 32 Bamboo Therapeutics, Inc. 33 BioMarin Pharmaceutical Inc. 34 Bristol-Myers Squibb Company 35 Capricor Therapeutics, Inc. 36 Catabasis Pharmaceuticals, Inc. 37 Daiichi Sankyo Company, Limited 38 Debiopharm International S.A. 39 Editas Medicine, Inc. 40 Eli Lilly and Company 41 EryDel SPA 42 FibroGen, Inc. 43 Galapagos NV 44 Genethon 45 GTx, Inc. 46 Idera Pharmaceuticals, Inc. 47 Italfarmaco S.p.A. 48 La Jolla Pharmaceutical Company 49 Marathon Pharmaceuticals, LLC 50 Marina Biotech, Inc. 51 Merck KGaA 52 Milo Biotechnology LLC 53 MyoTherix Inc. 54 NicOx S.A. 55 Nippon Shinyaku Co., Ltd. 56 nLife Therapeutics, S.L. 57 Nobelpharma Co., Ltd. 58 Pfizer Inc. 59 Prothelia, Inc. 60 PTC Therapeutics, Inc. 61 RASRx, LLC 62 ReveraGen BioPharma, Inc. 63 Sanofi 64 Santhera Pharmaceuticals Holding AG 65 Sarepta Therapeutics, Inc. 66 Summit Therapeutics Plc 67 Taiho Pharmaceutical Co., Ltd. 68 WAVE Life Sciences Ltd. 69 Zambon Company S.p.A. 70 Duchenne Muscular Dystrophy - Therapeutics Assessment 71 Assessment by Monotherapy Products 71 Assessment by Target 72 Assessment by Mechanism of Action 75 Assessment by Route of Administration 78 Assessment by Molecule Type 80 Drug Profiles 82 4-P001 - Drug Profile 82 AAV1-Foliistatin - Drug Profile 83 ACE-083 - Drug Profile 84 alisporivir - Drug Profile 85 Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 87 Antisense Oligonucleotide to Activate Dystrophin for Duchene Muscular Dystrophy - Drug Profile 88 Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 89 Antisense Oligonucleotide to Inhibit Myostatin for DMD and Becker MD - Drug Profile 90 Antisense Oligonucleotides to Inhibit ACVR2B for Duchenne Muscular Dystrophy - Drug Profile 91 Antisense RNAi Oligonucleotides for Duchenne Muscular Dystrophy - Drug Profile 92 Aptamer to Activate Calcineurin A for Genetic Disorders and Musculoskeletal Disorders - Drug Profile 93 arbekacin - Drug Profile 94 ARM-210 - Drug Profile 95 AT-300 - Drug Profile 96 ataluren - Drug Profile 98 Biostrophin - Drug Profile 102 BMBD-001 - Drug Profile 103 BMN-044 - Drug Profile 104 BMN-045 - Drug Profile 106 BMN-053 - Drug Profile 108 BMS-986089 - Drug Profile 110 CAP-1002 - Drug Profile 111 CAT-1004 - Drug Profile 113 CAT-1040 - Drug Profile 115 CAT-1041 - Drug Profile 116 DA-4210 - Drug Profile 117 deflazacort - Drug Profile 118 dexamethasone sodium phosphate - Drug Profile 119 domagrozumab - Drug Profile 121 drisapersen - Drug Profile 122 Drug for Duchenne Muscular Dystrophy - Drug Profile 124 Drugs for Duchenne Muscular Dystrophy - Drug Profile 125 DS-5141b - Drug Profile 126 DT-200 - Drug Profile 127 enobosarm - Drug Profile 129 eteplirsen - Drug Profile 131 FG-3019 - Drug Profile 134 Gene Therapy for Duchenne Muscular Dystrophy - Drug Profile 136 Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 137 Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 138 Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 139 Gene Therapy to Activate Dystrophine for Duchenne Muscular Dystrophy - Drug Profile 140 givinostat - Drug Profile 141 Gtx-027 - Drug Profile 143 IB-DMD - Drug Profile 144 idebenone - Drug Profile 145 IMO-8400 - Drug Profile 148 LJPC-30Sa - Drug Profile 150 LJPC-30Sb - Drug Profile 151 MG-53 - Drug Profile 152 MP-101 - Drug Profile 154 Myomir - Drug Profile 155 naproxcinod - Drug Profile 156 NBD Peptide - Drug Profile 158 NS-065 - Drug Profile 160 Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 161 Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 162 Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 163 Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 164 Oligonucleotides for Ischemic Heart Disease and Duchenne Muscular Dystrophy - Drug Profile 165 OSX-200 - Drug Profile 166 poloxamer - Drug Profile 167 PRO-052 - Drug Profile 169 PRO-055 - Drug Profile 171 PRT-01 - Drug Profile 173 Recombinant Protein for Muscular Dystrophy - Drug Profile 175 Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 176 Recombinant Proteins for Duchenne Muscular Dystrophy - Drug Profile 177 rimeporide - Drug Profile 178 RTC-13 - Drug Profile 179 Small Molecule for Duchenne Muscular Dystrophy - Drug Profile 181 Small Molecule for Duchenne Muscular Dystrophy - Drug Profile 182 Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 183 Small Molecules for Duchenne Muscular Dystrophy - Drug Profile 184 Small Molecules to Target Utrophin for Duchenne Muscular Dystrophy - Drug Profile 185 SMT-022357 - Drug Profile 186 SMTC-1100 - Drug Profile 187 SRP-4008 - Drug Profile 189 SRP-4044 - Drug Profile 190 SRP-4045 - Drug Profile 191 SRP-4050 - Drug Profile 192 SRP-4052 - Drug Profile 193 SRP-4053 - Drug Profile 194 SRP-4055 - Drug Profile 195 Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 196 Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 198 Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 199 Stryka-516 - Drug Profile 200 Stryka-969 - Drug Profile 201 StrykaPro-1 - Drug Profile 202 Synthetic Peptides for Duchene Muscle Dystrophy - Drug Profile 203 tadalafil - Drug Profile 204 TAS-205 - Drug Profile 206 TVN-102 - Drug Profile 207 TXA-127 - Drug Profile 208 Verolone - Drug Profile 211 ZP-049 - Drug Profile 213 Duchenne Muscular Dystrophy - Recent Pipeline Updates 214 Duchenne Muscular Dystrophy - Dormant Projects 279 Duchenne Muscular Dystrophy - Dormant Projects 279 Duchenne Muscular Dystrophy - Discontinued Products 282 Duchenne Muscular Dystrophy - Product Development Milestones 283 Featured News & Press Releases 283 Appendix 289 Methodology 289 Coverage 289 Secondary Research 289 Primary Research 289 Expert Panel Validation 289 Contact Us 289 Disclaimer 290

List of Tables Number of Products under Development for Duchenne Muscular Dystrophy, H1 2016 18 Number of Products under Development for Duchenne Muscular Dystrophy - Comparative Analysis, H1 2016 19 Number of Products under Development by Companies, H1 2016 21 Number of Products under Development by Companies, H1 2016 (Contd..1) 22 Number of Products under Development by Companies, H1 2016 (Contd..2) 23 Number of Products under Development by Companies, H1 2016 (Contd..3) 24 Number of Products under Investigation by Universities/Institutes, H1 2016 25 Comparative Analysis by Late Stage Development, H1 2016 26 Comparative Analysis by Clinical Stage Development, H1 2016 27 Comparative Analysis by Early Stage Development, H1 2016 28 Comparative Analysis by Unknown Stage Development, H1 2016 29 Products under Development by Companies, H1 2016 30 Products under Development by Companies, H1 2016 (Contd..1) 31 Products under Development by Companies, H1 2016 (Contd..2) 32 Products under Development by Companies, H1 2016 (Contd..3) 33 Products under Development by Companies, H1 2016 (Contd..4) 34 Products under Development by Companies, H1 2016 (Contd..5) 35 Products under Investigation by Universities/Institutes, H1 2016 36 Duchenne Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H1 2016 37 Duchenne Muscular Dystrophy - Pipeline by Anagenesis Biotechnology, H1 2016 38 Duchenne Muscular Dystrophy - Pipeline by Asklepios BioPharmaceutical, Inc., H1 2016 39 Duchenne Muscular Dystrophy - Pipeline by Bamboo Therapeutics, Inc., H1 2016 40 Duchenne Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 41 Duchenne Muscular Dystrophy - Pipeline by Bristol-Myers Squibb Company, H1 2016 42 Duchenne Muscular Dystrophy - Pipeline by Capricor Therapeutics, Inc., H1 2016 43 Duchenne Muscular Dystrophy - Pipeline by Catabasis Pharmaceuticals, Inc., H1 2016 44 Duchenne Muscular Dystrophy - Pipeline by Daiichi Sankyo Company, Limited, H1 2016 45 Duchenne Muscular Dystrophy - Pipeline by Debiopharm International S.A., H1 2016 46 Duchenne Muscular Dystrophy - Pipeline by Editas Medicine, Inc., H1 2016 47 Duchenne Muscular Dystrophy - Pipeline by Eli Lilly and Company, H1 2016 48 Duchenne Muscular Dystrophy - Pipeline by EryDel SPA, H1 2016 49 Duchenne Muscular Dystrophy - Pipeline by FibroGen, Inc., H1 2016 50 Duchenne Muscular Dystrophy - Pipeline by Galapagos NV, H1 2016 51 Duchenne Muscular Dystrophy - Pipeline by Genethon, H1 2016 52 Duchenne Muscular Dystrophy - Pipeline by GTx, Inc., H1 2016 53 Duchenne Muscular Dystrophy - Pipeline by Idera Pharmaceuticals, Inc., H1 2016 54 Duchenne Muscular Dystrophy - Pipeline by Italfarmaco S.p.A., H1 2016 55 Duchenne Muscular Dystrophy - Pipeline by La Jolla Pharmaceutical Company, H1 2016 56 Duchenne Muscular Dystrophy - Pipeline by Marathon Pharmaceuticals, LLC, H1 2016 57 Duchenne Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 58 Duchenne Muscular Dystrophy - Pipeline by Merck KGaA, H1 2016 59 Duchenne Muscular Dystrophy - Pipeline by Milo Biotechnology LLC, H1 2016 60 Duchenne Muscular Dystrophy - Pipeline by MyoTherix Inc., H1 2016 61 Duchenne Muscular Dystrophy - Pipeline by NicOx S.A., H1 2016 62 Duchenne Muscular Dystrophy - Pipeline by Nippon Shinyaku Co., Ltd., H1 2016 63 Duchenne Muscular Dystrophy - Pipeline by nLife Therapeutics, S.L., H1 2016 64 Duchenne Muscular Dystrophy - Pipeline by Nobelpharma Co., Ltd., H1 2016 65 Duchenne Muscular Dystrophy - Pipeline by Pfizer Inc., H1 2016 66 Duchenne Muscular Dystrophy - Pipeline by Prothelia, Inc., H1 2016 67 Duchenne Muscular Dystrophy - Pipeline by PTC Therapeutics, Inc., H1 2016 68 Duchenne Muscular Dystrophy - Pipeline by RASRx, LLC, H1 2016 69 Duchenne Muscular Dystrophy - Pipeline by ReveraGen BioPharma, Inc., H1 2016 70 Duchenne Muscular Dystrophy - Pipeline by Sanofi, H1 2016 71 Duchenne Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2016 72 Duchenne Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H1 2016 73 Duchenne Muscular Dystrophy - Pipeline by Summit Therapeutics Plc, H1 2016 74 Duchenne Muscular Dystrophy - Pipeline by Taiho Pharmaceutical Co., Ltd., H1 2016 75 Duchenne Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 76 Duchenne Muscular Dystrophy - Pipeline by Zambon Company S.p.A., H1 2016 77 Assessment by Monotherapy Products, H1 2016 78 Number of Products by Stage and Target, H1 2016 80 Number of Products by Stage and Mechanism of Action, H1 2016 83 Number of Products by Stage and Route of Administration, H1 2016 86 Number of Products by Stage and Molecule Type, H1 2016 88 Duchenne Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 221 Duchenne Muscular Dystrophy - Dormant Projects, H1 2016 286 Duchenne Muscular Dystrophy - Dormant Projects (Contd..1), H1 2016 287 Duchenne Muscular Dystrophy - Dormant Projects (Contd..2), H1 2016 288 Duchenne Muscular Dystrophy - Discontinued Products, H1 2016 289 List of Figures Number of Products under Development for Duchenne Muscular Dystrophy, H1 2016 18 Number of Products under Development for Duchenne Muscular Dystrophy - Comparative Analysis, H1 2016 19 Number of Products under Development by Companies, H1 2016 20 Number of Products under Investigation by Universities/Institutes, H1 2016 25 Comparative Analysis by Late Stage Development, H1 2016 26 Comparative Analysis by Clinical Stage Development, H1 2016 27 Comparative Analysis by Early Stage Products, H1 2016 28 Assessment by Monotherapy Products, H1 2016 78 Number of Products by Top 10 Targets, H1 2016 79 Number of Products by Stage and Top 10 Targets, H1 2016 79 Number of Products by Top 10 Mechanism of Actions, H1 2016 82 Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 82 Number of Products by Routes of Administration, H1 2016 85 Number of Products by Stage and Routes of Administration, H1 2016 85 Number of Products by Top 10 Molecule Types, H1 2016 87 Number of Products by Stage and Molecule Types, H1 2016 87

Acceleron Pharma, Inc. Anagenesis Biotechnology Asklepios BioPharmaceutical, Inc. Bamboo Therapeutics, Inc. BioMarin Pharmaceutical Inc. Bristol-Myers Squibb Company Capricor Therapeutics, Inc. Catabasis Pharmaceuticals, Inc. Daiichi Sankyo Company, Limited Debiopharm International S.A. Editas Medicine, Inc. Eli Lilly and Company EryDel SPA FibroGen, Inc. Galapagos NV Genethon GTx, Inc. Idera Pharmaceuticals, Inc. Italfarmaco S.p.A. La Jolla Pharmaceutical Company Marathon Pharmaceuticals, LLC Marina Biotech, Inc. Merck KGaA Milo Biotechnology LLC MyoTherix Inc. NicOx S.A. Nippon Shinyaku Co., Ltd. nLife Therapeutics, S.L. Nobelpharma Co., Ltd. Pfizer Inc. Prothelia, Inc. PTC Therapeutics, Inc. RASRx, LLC ReveraGen BioPharma, Inc. Sanofi Santhera Pharmaceuticals Holding AG Sarepta Therapeutics, Inc. Summit Therapeutics Plc Taiho Pharmaceutical Co., Ltd. WAVE Life Sciences Ltd. Zambon Company S.p.A.