筋緊張性ジストロフィー(Myotonic Dystrophy)の治療薬パイプライン動向（2015年上半期版）...市場調査レポートについてご紹介

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Myotonic Dystrophy Overview 7
Therapeutics Development 8
Pipeline Products for Myotonic Dystrophy – Overview 8
Pipeline Products for Myotonic Dystrophy – Comparative Analysis 9
Myotonic Dystrophy – Therapeutics under Development by Companies 10
Myotonic Dystrophy – Therapeutics under Investigation by Universities/Institutes 11
Myotonic Dystrophy – Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Myotonic Dystrophy – Products under Development by Companies 14
Myotonic Dystrophy – Products under Investigation by Universities/Institutes 16
Myotonic Dystrophy – Companies Involved in Therapeutics Development 17
F. Hoffmann-La Roche Ltd. 17
Genzyme Corporation 18
Isis Pharmaceuticals, Inc. 19
Marina Biotech, Inc. 20
Prosensa Therapeutics B.V. 21
Sarepta Therapeutics, Inc. 22
Valentia Biopharma S.L. 23
Myotonic Dystrophy – Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Target 25
Assessment by Mechanism of Action 27
Assessment by Route of Administration 29
Assessment by Molecule Type 31
Drug Profiles 33
Antisense Oligonucleotide for Myotonic Dystrophy – Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy – Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
ISIS-DMPKRx – Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
PRO-135 – Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
RNAi Oligonucleotide for Myotonic Dystrophy – Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Small Molecule to Target RNA for Myotonic Dystrophy – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Small Molecules for Myotonic Dystrophy Type 1 – Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Small Molecules to Target CUG RNA for Myotonic Dystrophy 1 – Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Small Molecules to Target RNA for Myotonic Dystrophy – Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
SRT-149 – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
SRT-152 – Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
VAL-0411 – Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
VLT-001 – Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
VLT-002 – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
VLT-003 – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
VLT-004 – Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
VLT-005 – Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
VLT-014 – Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
VLT-015 – Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
VLT-016 – Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
VLT-017 – Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
VLT-025 – Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Myotonic Dystrophy – Recent Pipeline Updates 56
Myotonic Dystrophy – Product Development Milestones 57
Featured News & Press Releases 57
Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015 57
Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 57
Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 58
Appendix 60
Methodology 60
Coverage 60
Secondary Research 60
Primary Research 60
Expert Panel Validation 60
Contact Us 61
Disclaimer 61