Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 5 |
| List of Tables | 6 | 1 |
| List of Figures | 6 | 1 |
| Introduction | 7 | 1 |
| Global Markets Direct Report Coverage | 7 | 1 |
| Dystrophin (DMD) Overview | 8 | 1 |
| Therapeutics Development | 9 | 3 |
| Dystrophin (DMD) Products under Development by Stage of Development | 9 | 1 |
| Dystrophin (DMD) Products under Development by Therapy Area | 10 | 1 |
| Dystrophin (DMD) Products under Development by Indication | 11 | 1 |
| Dystrophin (DMD) Pipeline Products Glance | 12 | 2 |
| Late Stage Products | 12 | 1 |
| Early Stage Products | 13 | 1 |
| Dystrophin (DMD) Products under Development by Companies | 14 | 2 |
| Dystrophin (DMD) Products under Development by Universities/Institutes | 16 | 2 |
| Dystrophin (DMD) Therapeutics Assessment | 18 | 6 |
| Assessment by Monotherapy/Combination Products | 18 | 1 |
| Assessment by Mechanism of Action | 19 | 1 |
| Assessment by Route of Administration | 20 | 2 |
| Assessment by Molecule Type | 22 | 2 |
| Dystrophin (DMD) Companies Involved in Therapeutics Development | 24 | 8 |
| Asklepios BioPharmaceutical, Inc. | 24 | 1 |
| BioMarin Pharmaceutical Inc. | 25 | 1 |
| Daiichi Sankyo Company, Limited | 26 | 1 |
| Genethon | 27 | 1 |
| Nippon Shinyaku Co., Ltd. | 28 | 1 |
| PTC Therapeutics, Inc. | 29 | 1 |
| Sarepta Therapeutics, Inc. | 30 | 1 |
| WAVE Life Sciences Ltd. | 31 | 1 |
| Dystrophin (DMD) Drug Profiles | 32 | 37 |
| Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 32 | 1 |
| Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 33 | 1 |
| Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 34 | 1 |
| Biostrophin Drug Profile | 35 | 1 |
| BMBD-001 Drug Profile | 36 | 1 |
| DS-5141 Drug Profile | 37 | 1 |
| eteplirsen Drug Profile | 38 | 11 |
| Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 49 | 1 |
| Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 50 | 1 |
| Gene Therapy to Activate Dystrophin for Muscular Dystrophy Drug Profile | 51 | 1 |
| NS-065 Drug Profile | 52 | 1 |
| Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 53 | 1 |
| Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 54 | 1 |
| Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 55 | 1 |
| Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 56 | 1 |
| PRO-055 Drug Profile | 57 | 2 |
| SGT-001 Drug Profile | 59 | 1 |
| Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 60 | 1 |
| SRP-4008 Drug Profile | 61 | 1 |
| SRP-4044 Drug Profile | 62 | 1 |
| SRP-4045 Drug Profile | 63 | 1 |
| SRP-4050 Drug Profile | 64 | 1 |
| SRP-4052 Drug Profile | 65 | 1 |
| SRP-4053 Drug Profile | 66 | 1 |
| SRP-4055 Drug Profile | 67 | 1 |
| Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy Drug Profile | 68 | 1 |
| Dystrophin (DMD) Dormant Projects | 69 | 1 |
| Dystrophin (DMD) Discontinued Products | 70 | 1 |
| Dystrophin (DMD) Featured News &Press Releases | 71 | 9 |
| Jun 06, 2016: Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA | 71 | 1 |
| May 25, 2016: Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date | 71 | 1 |
| Apr 25, 2016: MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug | 71 | 1 |
| Apr 25, 2016: Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy | 72 | 1 |
| Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen | 73 | 1 |
| Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy | 73 | 1 |
| Feb 25, 2016: Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment | 73 | 1 |
| Feb 08, 2016: Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen | 74 | 1 |
| Jan 20, 2016: FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area | 74 | 1 |
| Dec 18, 2015: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | 75 | 1 |
| Nov 17, 2015: Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology | 75 | 1 |
| Oct 22, 2015: Gene Therapy Treats All Muscles in the Body in Muscular Dystrophy Dogs | 76 | 1 |
| Oct 14, 2015: Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy | 77 | 1 |
| Oct 01, 2015: Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy | 77 | 2 |
| Aug 25, 2015: Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping | 79 | 1 |
| Appendix | 80 | 2 |
| Methodology | 80 | 1 |
| Coverage | 80 | 1 |
| Secondary Research | 80 | 1 |
| Primary Research | 80 | 1 |
| Expert Panel Validation | 80 | 1 |
| Contact Us | 80 | 1 |
| Disclaimer | 81 | 1 |
