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Published: May, 2015 | Pages:
121 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Muscular Dystrophy - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Muscular Dystrophy - Pipeline Review, H1 2015', provides an overview of the Muscular Dystrophy's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Muscular Dystrophy, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Muscular Dystrophy and special features on late-stage and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Muscular Dystrophy - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Muscular Dystrophy and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Muscular Dystrophy products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Muscular Dystrophy pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Muscular Dystrophy - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Muscular Dystrophy pipeline depth and focus of Indication therapeutics - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and Scope - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents
Table of Contents 2 List of Tables 4 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Muscular Dystrophy Overview 7 Therapeutics Development 8 Pipeline Products for Muscular Dystrophy - Overview 8 Pipeline Products for Muscular Dystrophy - Comparative Analysis 9 Muscular Dystrophy - Therapeutics under Development by Companies 10 Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 12 Muscular Dystrophy - Pipeline Products Glance 13 Late Stage Products 13 Clinical Stage Products 14 Early Stage Products 15 Muscular Dystrophy - Products under Development by Companies 16 Muscular Dystrophy - Products under Investigation by Universities/Institutes 19 Muscular Dystrophy - Companies Involved in Therapeutics Development 20 Acceleron Pharma, Inc. 20 aTyr Pharma, Inc. 21 Benitec Biopharma Limited 22 Bioblast Pharma Ltd. 23 BioMarin Pharmaceutical Inc. 24 Evotec AG 25 F. Hoffmann-La Roche Ltd. 26 Fate Therapeutics, Inc. 27 Genethon 28 Genzyme Corporation 29 Isis Pharmaceuticals, Inc. 30 Marina Biotech, Inc. 31 Novogen Limited 32 PolyNovo Limited 33 Prothelia, Inc. 34 SanBio, Inc. 35 Santhera Pharmaceuticals Holding AG 36 Sarepta Therapeutics, Inc. 37 Selecta Biosciences, Inc. 38 Takeda Pharmaceutical Company Limited 39 Valentia Biopharma S.L. 40 Muscular Dystrophy - Therapeutics Assessment 41 Assessment by Monotherapy Products 41 Assessment by Target 42 Assessment by Mechanism of Action 44 Assessment by Route of Administration 46 Assessment by Molecule Type 48 Drug Profiles 50 ACE-083 - Drug Profile 50 Antisense Oligonucleotide for Myotonic Dystrophy - Drug Profile 51 Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile 52 AOD-9604 - Drug Profile 53 ATYR-1940 - Drug Profile 55 Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 56 Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 57 Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 58 Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 59 ISIS-DMPK-2.5 - Drug Profile 60 ketoprofen - Drug Profile 61 omigapil - Drug Profile 62 Pabparna - Drug Profile 64 PRO-135 - Drug Profile 65 PRT-01 - Drug Profile 66 PRT-300 - Drug Profile 67 Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 68 RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 69 RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 70 SB-308 - Drug Profile 71 Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 72 Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 73 Small Molecules for CNS and Muscular Dystrophy Diseases - Drug Profile 74 Small Molecules for Dysferlinopathies - Drug Profile 75 Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 76 Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 78 SRT-149 - Drug Profile 79 SRT-152 - Drug Profile 80 Stem Cell Therapy for Muscular Dystrophy - Drug Profile 81 Stem Cell Therapy for Musculoskeletal Disorders - Drug Profile 82 Stryka-232 - Drug Profile 83 Stryka-234 - Drug Profile 84 Stryka-425 - Drug Profile 85 Stryka-533 - Drug Profile 86 Stryka-978 - Drug Profile 87 trehalose - Drug Profile 88 VAL-0411 - Drug Profile 89 VLT-001 - Drug Profile 90 VLT-002 - Drug Profile 91 VLT-003 - Drug Profile 92 VLT-004 - Drug Profile 93 VLT-005 - Drug Profile 94 VLT-014 - Drug Profile 95 VLT-015 - Drug Profile 96 VLT-016 - Drug Profile 97 VLT-017 - Drug Profile 98 VLT-025 - Drug Profile 99 Muscular Dystrophy - Recent Pipeline Updates 100 Muscular Dystrophy - Dormant Projects 106 Muscular Dystrophy - Discontinued Products 107 Muscular Dystrophy - Product Development Milestones 108 Featured News & Press Releases 108 Appendix 116 Methodology 116 Coverage 116 Secondary Research 116 Primary Research 116 Expert Panel Validation 116 Contact Us 116 Disclaimer 117
List of Tables Number of Products under Development for Muscular Dystrophy, H1 2015 12 Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H1 2015 13 Number of Products under Development by Companies, H1 2015 14 Number of Products under Development by Companies, H1 2015 (Contd..1) 15 Number of Products under Investigation by Universities/Institutes, H1 2015 16 Comparative Analysis by Late Stage Development, H1 2015 17 Comparative Analysis by Clinical Stage Development, H1 2015 18 Comparative Analysis by Early Stage Development, H1 2015 19 Products under Development by Companies, H1 2015 20 Products under Development by Companies, H1 2015 (Contd..1) 21 Products under Development by Companies, H1 2015 (Contd..2) 22 Products under Investigation by Universities/Institutes, H1 2015 23 Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H1 2015 24 Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H1 2015 25 Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H1 2015 26 Muscular Dystrophy - Pipeline by Bioblast Pharma Ltd., H1 2015 27 Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2015 28 Muscular Dystrophy - Pipeline by Evotec AG, H1 2015 29 Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2015 30 Muscular Dystrophy - Pipeline by Fate Therapeutics, Inc., H1 2015 31 Muscular Dystrophy - Pipeline by Genethon, H1 2015 32 Muscular Dystrophy - Pipeline by Genzyme Corporation, H1 2015 33 Muscular Dystrophy - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 34 Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H1 2015 35 Muscular Dystrophy - Pipeline by Novogen Limited, H1 2015 36 Muscular Dystrophy - Pipeline by PolyNovo Limited, H1 2015 37 Muscular Dystrophy - Pipeline by Prothelia, Inc., H1 2015 38 Muscular Dystrophy - Pipeline by SanBio, Inc., H1 2015 39 Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2015 40 Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H1 2015 41 Muscular Dystrophy - Pipeline by Selecta Biosciences, Inc., H1 2015 42 Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H1 2015 43 Muscular Dystrophy - Pipeline by Valentia Biopharma S.L., H1 2015 44 Assessment by Monotherapy Products, H1 2015 45 Number of Products by Stage and Target, H1 2015 47 Number of Products by Stage and Mechanism of Action, H1 2015 49 Number of Products by Stage and Route of Administration, H1 2015 51 Number of Products by Stage and Molecule Type, H1 2015 53 Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H1 2015 104 Muscular Dystrophy - Dormant Projects, H1 2015 110 Muscular Dystrophy - Discontinued Products, H1 2015 111
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