Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 9 |
| List of Tables | 9 | 1 |
| List of Figures | 10 | 1 |
| Introduction | 11 | 1 |
| Global Markets Direct Report Coverage | 11 | 1 |
| Muscular Dystrophy Overview | 12 | 1 |
| Therapeutics Development | 13 | 2 |
| Pipeline Products for Muscular Dystrophy Overview | 13 | 1 |
| Pipeline Products for Muscular Dystrophy Comparative Analysis | 14 | 1 |
| Muscular Dystrophy Therapeutics under Development by Companies | 15 | 2 |
| Muscular Dystrophy Therapeutics under Investigation by Universities/Institutes | 17 | 1 |
| Muscular Dystrophy Pipeline Products Glance | 18 | 3 |
| Late Stage Products | 18 | 1 |
| Clinical Stage Products | 19 | 1 |
| Early Stage Products | 20 | 1 |
| Muscular Dystrophy Products under Development by Companies | 21 | 3 |
| Muscular Dystrophy Products under Investigation by Universities/Institutes | 24 | 1 |
| Muscular Dystrophy Companies Involved in Therapeutics Development | 25 | 24 |
| Acceleron Pharma, Inc. | 25 | 1 |
| AMO Pharma Limited | 26 | 1 |
| Asahi Kasei Pharma Corp. | 27 | 1 |
| aTyr Pharma, Inc. | 28 | 1 |
| Benitec Biopharma Limited | 29 | 1 |
| Bio Blast Pharma Ltd. | 30 | 1 |
| Biophytis SAS | 31 | 1 |
| Corcept Therapeutics Incorporated | 32 | 1 |
| Evotec AG | 33 | 1 |
| F. Hoffmann-La Roche Ltd. | 34 | 1 |
| Genethon | 35 | 1 |
| Ionis Pharmaceuticals, Inc. | 36 | 1 |
| Marina Biotech, Inc. | 37 | 1 |
| Medestea Research &Production S.p.A. | 38 | 1 |
| Novogen Limited | 39 | 1 |
| Pfizer Inc. | 40 | 1 |
| Prothelia, Inc. | 41 | 1 |
| SanBio, Inc. | 42 | 1 |
| Santhera Pharmaceuticals Holding AG | 43 | 1 |
| Sarepta Therapeutics, Inc. | 44 | 1 |
| Selecta Biosciences, Inc. | 45 | 1 |
| Strykagen Corporation | 46 | 1 |
| Takeda Pharmaceutical Company Limited | 47 | 1 |
| WAVE Life Sciences Ltd. | 48 | 1 |
| Muscular Dystrophy Therapeutics Assessment | 49 | 9 |
| Assessment by Monotherapy Products | 49 | 1 |
| Assessment by Target | 50 | 2 |
| Assessment by Mechanism of Action | 52 | 2 |
| Assessment by Route of Administration | 54 | 2 |
| Assessment by Molecule Type | 56 | 2 |
| Drug Profiles | 58 | 67 |
| ACE-083 Drug Profile | 58 | 2 |
| Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy Drug Profile | 60 | 1 |
| Antisense RNAi Oligonucleotides for Myotonic Dystrophy Drug Profile | 61 | 1 |
| ATYR-1940 Drug Profile | 62 | 3 |
| baliforsen Drug Profile | 65 | 1 |
| BIO-103 Drug Profile | 66 | 1 |
| domagrozumab Drug Profile | 67 | 2 |
| Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A Drug Profile | 69 | 1 |
| elcatonin Drug Profile | 70 | 1 |
| Gene Therapy for Muscular Dystrophy and Liver Diseases Drug Profile | 71 | 1 |
| Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies Drug Profile | 72 | 1 |
| Gene Therapy to Activate Dysferlin for Dysferlinopathies Drug Profile | 73 | 1 |
| Gene Therapy to Activate Dystrophin for Muscular Dystrophy Drug Profile | 74 | 1 |
| IUCT-169 Drug Profile | 75 | 1 |
| IUCT-290 Drug Profile | 76 | 1 |
| IUCT-309 Drug Profile | 77 | 1 |
| ketoprofen Drug Profile | 78 | 1 |
| LR-08 Drug Profile | 79 | 1 |
| MED-1101 Drug Profile | 80 | 1 |
| mexiletine hydrochloride Drug Profile | 81 | 1 |
| Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy Drug Profile | 82 | 1 |
| Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy Drug Profile | 83 | 1 |
| omigapil Drug Profile | 84 | 2 |
| Pabparna Drug Profile | 86 | 1 |
| poloxamer Drug Profile | 87 | 2 |
| PRT-01 Drug Profile | 89 | 2 |
| Recombinant Protein to Activate Utrophin for Muscular Dystrophies Drug Profile | 91 | 1 |
| RNAi Gene Therapy to Inhibit Myotilin for LGMD Drug Profile | 92 | 1 |
| RP-33 Drug Profile | 93 | 1 |
| SB-308 Drug Profile | 94 | 1 |
| SIWA-318 Drug Profile | 95 | 1 |
| Small Molecule to Inhibit DUX4 for Muscular Dystrophy Drug Profile | 96 | 1 |
| Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 Drug Profile | 97 | 1 |
| Small Molecule to Target RNA for Myotonic Dystrophy Drug Profile | 98 | 1 |
| Small Molecules for Dysferlinopathies Drug Profile | 99 | 1 |
| Small Molecules for Facioscapulohumeral Muscular Dystrophy Drug Profile | 100 | 1 |
| Small Molecules for Myotonic Dystrophy Drug Profile | 101 | 1 |
| Small Molecules for Myotonic Dystrophy Type 1 Drug Profile | 102 | 1 |
| Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy Drug Profile | 103 | 1 |
| Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular Dystrophy Drug Profile | 104 | 1 |
| Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I Drug Profile | 105 | 1 |
| Small Molecules to Target RNA for Myotonic Dystrophy Drug Profile | 106 | 1 |
| SRT-149 Drug Profile | 107 | 1 |
| SRT-152 Drug Profile | 108 | 1 |
| Stem Cell Therapy for Muscular Dystrophy Drug Profile | 109 | 1 |
| Stryka-232 Drug Profile | 110 | 1 |
| Stryka-234 Drug Profile | 111 | 1 |
| Stryka-425 Drug Profile | 112 | 1 |
| Stryka-533 Drug Profile | 113 | 1 |
| Stryka-978 Drug Profile | 114 | 1 |
| tideglusib Drug Profile | 115 | 1 |
| trehalose Drug Profile | 116 | 4 |
| TXA-127 Drug Profile | 120 | 3 |
| VAL-0411 Drug Profile | 123 | 1 |
| VAL-1205 Drug Profile | 124 | 1 |
| Muscular Dystrophy Dormant Projects | 125 | 2 |
| Muscular Dystrophy Discontinued Products | 127 | 1 |
| Muscular Dystrophy Product Development Milestones | 128 | 15 |
| Featured News &Press Releases | 128 | 1 |
| Oct 13, 2016: Acceleron to Host Educational Webcast on Facioscapulohumeral Muscular Dystrophy (FSHD) with Leading Researcher and Provide Overview of ACE-083 Phase 2 Trial | 128 | 1 |
| Sep 26, 2016: Bioblast's Special Medical Advisor to Make a Presentation Today On Trehalose as a Potential Therapeutic Agent for OPMD | 128 | 1 |
| Sep 12, 2016: Bioblast Pharma Reports Results of a Clinical Trial of Trehalose in Oculopharyngeal Muscular Dystrophy (OPMD) Patients | 129 | 1 |
| Aug 30, 2016: Santhera Receives FDA Grant in Support of its Ongoing Phase I Trial with Omigapil in Congenital Muscular Dystrophy | 130 | 1 |
| Aug 10, 2016: AMO Pharma Initiates Phase 2 Clinical Trial in UK for AMO-02 for Treatment of Congenital and Juvenile Onset Myotonic Dystrophy | 131 | 1 |
| Jul 08, 2016: Acceleron Announces ACE-083 Phase 1 Results at the 14th International Congress on Neuromuscular Diseases | 132 | 1 |
| Jun 29, 2016: Acceleron Announces Presentation of ACE-083 Phase 1 Healthy Volunteer Data at the 14th International Congress on Neuromuscular Disease | 133 | 1 |
| Jun 09, 2016: Bioblast Pharma Updates Corporate Plans | 133 | 1 |
| Apr 21, 2016: Benitec to present data on muscular dystrophy program at the ASGCT 2016 Annual Meeting | 134 | 1 |
| Apr 14, 2016: BioBlast Pharma to Present Final HOPEMD Phase 2 Clinical Study Results of Trehalose in Oculopharyngeal Muscular Dystrophy at the American Academy of Neurology (AAN) 2016 Annual Meeting | 135 | 1 |
| Mar 16, 2016: BioBlast Pharma Announces Positive Final Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in Oculopharyngeal Muscular Dystrophy | 135 | 2 |
| Feb 17, 2016: Tarix Orphan Granted Orphan Drug Status for TXA127 as Potential Treatment for Congenital Muscular Dystrophy (MDC1A) | 137 | 1 |
| Dec 09, 2015: BioBlast Pharma Provides Research and Development Update | 138 | 1 |
| Dec 01, 2015: Acceleron Announces Oral Plenary Presentation for ACE-083 in the Late Breaking Clinical Trials Session at the 8th International Conference on Cachexia, Sarcopenia and Muscle Wasting | 138 | 1 |
| Oct 27, 2015: BioBlast Announces Positive Interim Results From HOPEMD Phase 2 Clinical Study of Cabaletta in Oculopharyngeal Muscular Dystrophy | 139 | 4 |
| Appendix | 143 | 2 |
| Methodology | 143 | 1 |
| Coverage | 143 | 1 |
| Secondary Research | 143 | 1 |
| Primary Research | 143 | 1 |
| Expert Panel Validation | 143 | 1 |
| Contact Us | 143 | 1 |
| Disclaimer | 144 | 1 |
