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Published: Nov, 2016 | Pages:
144 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Muscular Dystrophy - Pipeline Review, H2 2016 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Muscular Dystrophy - Pipeline Review, H2 2016, provides an overview of the Muscular Dystrophy (Musculoskeletal Disorders) pipeline landscape. Muscular dystrophy is a group of diseases in which muscle fibers are unusually susceptible to damage. These damaged muscles become progressively weaker. Symptoms usually appear before age 6 and may appear as early as infancy. They may include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Muscular Dystrophy - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 5, 2, 24 and 14 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 3 and 4 molecules, respectively.Muscular Dystrophy. Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Scope - The pipeline guide provides a snapshot of the global therapeutic landscape of Muscular Dystrophy (Musculoskeletal Disorders). - The pipeline guide reviews pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. - The pipeline guide reviews key companies involved in Muscular Dystrophy (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Muscular Dystrophy (Musculoskeletal Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. - The pipeline guide encapsulates all the dormant and discontinued pipeline projects. - The pipeline guide reviews latest news related to pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders) Reasons To Buy - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. - Find and recognize significant and varied types of therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders). - Classify potential new clients or partners in the target demographic. - Develop tactical initiatives by understanding the focus areas of leading companies. - Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics. - Formulate corrective measures for pipeline projects by understanding Muscular Dystrophy (Musculoskeletal Disorders) 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. - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Muscular Dystrophy Overview 8 Therapeutics Development 9 Pipeline Products for Muscular Dystrophy - Overview 9 Pipeline Products for Muscular Dystrophy - Comparative Analysis 10 Muscular Dystrophy - Therapeutics under Development by Companies 11 Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 13 Muscular Dystrophy - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Muscular Dystrophy - Products under Development by Companies 17 Muscular Dystrophy - Products under Investigation by Universities/Institutes 20 Muscular Dystrophy - Companies Involved in Therapeutics Development 21 Acceleron Pharma, Inc. 21 AMO Pharma Limited 22 Asahi Kasei Pharma Corp. 23 aTyr Pharma, Inc. 24 Benitec Biopharma Limited 25 Bio Blast Pharma Ltd. 26 Biophytis SAS 27 Corcept Therapeutics Incorporated 28 Evotec AG 29 F. Hoffmann-La Roche Ltd. 30 Genethon 31 Ionis Pharmaceuticals, Inc. 32 Marina Biotech, Inc. 33 Medestea Research & Production S.p.A. 34 Novogen Limited 35 Pfizer Inc. 36 Prothelia, Inc. 37 SanBio, Inc. 38 Santhera Pharmaceuticals Holding AG 39 Sarepta Therapeutics, Inc. 40 Selecta Biosciences, Inc. 41 Strykagen Corporation 42 Takeda Pharmaceutical Company Limited 43 WAVE Life Sciences Ltd. 44 Muscular Dystrophy - Therapeutics Assessment 45 Assessment by Monotherapy Products 45 Assessment by Target 46 Assessment by Mechanism of Action 48 Assessment by Route of Administration 50 Assessment by Molecule Type 52 Drug Profiles 54 ACE-083 - Drug Profile 54 Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 56 Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 57 ATYR-1940 - Drug Profile 58 baliforsen - Drug Profile 61 BIO-103 - Drug Profile 62 domagrozumab - Drug Profile 63 Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 65 elcatonin - Drug Profile 66 Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 67 Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 68 Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 69 Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 70 IUCT-169 - Drug Profile 71 IUCT-290 - Drug Profile 72 IUCT-309 - Drug Profile 73 ketoprofen - Drug Profile 74 LR-08 - Drug Profile 75 MED-1101 - Drug Profile 76 mexiletine hydrochloride - Drug Profile 77 Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 78 Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 79 omigapil - Drug Profile 80 Pabparna - Drug Profile 82 poloxamer - Drug Profile 83 PRT-01 - Drug Profile 85 Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 87 RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 88 RP-33 - Drug Profile 89 SB-308 - Drug Profile 90 SIWA-318 - Drug Profile 91 Small Molecule to Inhibit DUX4 for Muscular Dystrophy - Drug Profile 92 Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 93 Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 94 Small Molecules for Dysferlinopathies - Drug Profile 95 Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 96 Small Molecules for Myotonic Dystrophy - Drug Profile 97 Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 98 Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 99 Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular Dystrophy - Drug Profile 100 Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 101 Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 102 SRT-149 - Drug Profile 103 SRT-152 - Drug Profile 104 Stem Cell Therapy for Muscular Dystrophy - Drug Profile 105 Stryka-232 - Drug Profile 106 Stryka-234 - Drug Profile 107 Stryka-425 - Drug Profile 108 Stryka-533 - Drug Profile 109 Stryka-978 - Drug Profile 110 tideglusib - Drug Profile 111 trehalose - Drug Profile 112 TXA-127 - Drug Profile 116 VAL-0411 - Drug Profile 119 VAL-1205 - Drug Profile 120 Muscular Dystrophy - Dormant Projects 121 Muscular Dystrophy - Discontinued Products 123 Muscular Dystrophy - Product Development Milestones 124 Featured News & Press Releases 124 Appendix 139 Methodology 139 Coverage 139 Secondary Research 139 Primary Research 139 Expert Panel Validation 139 Contact Us 139 Disclaimer 140
List of Tables
Number of Products under Development for Muscular Dystrophy, H2 2016 13 Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H2 2016 14 Number of Products under Development by Companies, H2 2016 15 Number of Products under Development by Companies, H2 2016 (Contd..1) 16 Number of Products under Investigation by Universities/Institutes, H2 2016 17 Comparative Analysis by Late Stage Development, H2 2016 18 Comparative Analysis by Clinical Stage Development, H2 2016 19 Comparative Analysis by Early Stage Development, H2 2016 20 Products under Development by Companies, H2 2016 21 Products under Development by Companies, H2 2016 (Contd..1) 22 Products under Development by Companies, H2 2016 (Contd..2) 23 Products under Investigation by Universities/Institutes, H2 2016 24 Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H2 2016 25 Muscular Dystrophy - Pipeline by AMO Pharma Limited, H2 2016 26 Muscular Dystrophy - Pipeline by Asahi Kasei Pharma Corp., H2 2016 27 Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H2 2016 28 Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H2 2016 29 Muscular Dystrophy - Pipeline by Bio Blast Pharma Ltd., H2 2016 30 Muscular Dystrophy - Pipeline by Biophytis SAS, H2 2016 31 Muscular Dystrophy - Pipeline by Corcept Therapeutics Incorporated, H2 2016 32 Muscular Dystrophy - Pipeline by Evotec AG, H2 2016 33 Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H2 2016 34 Muscular Dystrophy - Pipeline by Genethon, H2 2016 35 Muscular Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 36 Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H2 2016 37 Muscular Dystrophy - Pipeline by Medestea Research & Production S.p.A., H2 2016 38 Muscular Dystrophy - Pipeline by Novogen Limited, H2 2016 39 Muscular Dystrophy - Pipeline by Pfizer Inc., H2 2016 40 Muscular Dystrophy - Pipeline by Prothelia, Inc., H2 2016 41 Muscular Dystrophy - Pipeline by SanBio, Inc., H2 2016 42 Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H2 2016 43 Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H2 2016 44 Muscular Dystrophy - Pipeline by Selecta Biosciences, Inc., H2 2016 45 Muscular Dystrophy - Pipeline by Strykagen Corporation, H2 2016 46 Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H2 2016 47 Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H2 2016 48 Assessment by Monotherapy Products, H2 2016 49 Number of Products by Stage and Target, H2 2016 51 Number of Products by Stage and Mechanism of Action, H2 2016 53 Number of Products by Stage and Route of Administration, H2 2016 55 Number of Products by Stage and Molecule Type, H2 2016 57 Muscular Dystrophy - Dormant Projects, H2 2016 125 Muscular Dystrophy - Dormant Projects (Contd..1), H2 2016 126 Muscular Dystrophy - Discontinued Products, H2 2016 127
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