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Myotonic Dystrophy - Pipeline Review, H1 2016

Published: Feb, 2016 | Pages: 63 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Myotonic Dystrophy - Pipeline Review, H1 2016

Summary
Global Markets Direct's, 'Myotonic Dystrophy - Pipeline Review, H1 2016', provides an overview of the Myotonic Dystrophy pipeline landscape. The report provides comprehensive information on the therapeutics under development for Myotonic Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Myotonic Dystrophy and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. The report 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. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data. Scope - The report provides a snapshot of the global therapeutic landscape of Myotonic Dystrophy - The report reviews pipeline therapeutics for Myotonic Dystrophy by companies and universities/research institutes based on information derived from company and industry-specific sources - The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages - The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved Myotonic Dystrophy therapeutics and enlists all their major and minor projects - The report assesses Myotonic Dystrophy therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type - The report summarizes all the dormant and discontinued pipeline projects - The report reviews latest news related to pipeline therapeutics for Myotonic Dystrophy Reasons to buy
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Identify and understand important and diverse types of therapeutics under development for Myotonic Dystrophy - Identify potential new clients or partners in the target demographic - Develop strategic initiatives by understanding the focus areas of leading companies - Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics - Devise corrective measures for pipeline projects by understanding Myotonic 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 6 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Myotonic Dystrophy Overview 8 Therapeutics Development 9 Pipeline Products for Myotonic Dystrophy - Overview 9 Pipeline Products for Myotonic Dystrophy - Comparative Analysis 10 Myotonic Dystrophy - Therapeutics under Development by Companies 11 Myotonic Dystrophy - Therapeutics under Investigation by Universities/Institutes 12 Myotonic Dystrophy - Pipeline Products Glance 13 Clinical Stage Products 13 Early Stage Products 14 Myotonic Dystrophy - Products under Development by Companies 15 Myotonic Dystrophy - Products under Investigation by Universities/Institutes 16 Myotonic Dystrophy - Companies Involved in Therapeutics Development 17 AMO Pharma Limited 17 BioMarin Pharmaceutical Inc. 18 F. Hoffmann-La Roche Ltd. 19 Ionis Pharmaceuticals, Inc. 20 Marina Biotech, Inc. 21 Valentia Biopharma S.L. 22 WAVE Life Sciences Ltd. 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 AMO-02 - Drug Profile 33 Product Description 33 Mechanism of Action 33 R&D Progress 33 Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 IONIS-DMPK2.5Rx - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 PRO-135 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 Small Molecules for Myotonic Dystrophy Type 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 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 SRT-149 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 SRT-152 - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 VAL-0411 - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 VLT-001 - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 VLT-002 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 VLT-003 - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 VLT-004 - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 VLT-005 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 VLT-014 - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 VLT-015 - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 VLT-016 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 VLT-017 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 VLT-025 - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Myotonic Dystrophy - Recent Pipeline Updates 57 Myotonic Dystrophy - Dormant Projects 58 Myotonic Dystrophy - Product Development Milestones 59 Featured News & Press Releases 59 Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015 59 Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 59 Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 60 Appendix 62 Methodology 62 Coverage 62 Secondary Research 62 Primary Research 62 Expert Panel Validation 62 Contact Us 62 Disclaimer 63
List of Tables
Number of Products under Development for Myotonic Dystrophy, H1 2016 9 Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H1 2016 10 Number of Products under Development by Companies, H1 2016 11 Number of Products under Investigation by Universities/Institutes, H1 2016 12 Comparative Analysis by Clinical Stage Development, H1 2016 13 Comparative Analysis by Early Stage Development, H1 2016 14 Products under Development by Companies, H1 2016 15 Products under Investigation by Universities/Institutes, H1 2016 16 Myotonic Dystrophy - Pipeline by AMO Pharma Limited, H1 2016 17 Myotonic Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 18 Myotonic Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 19 Myotonic Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 20 Myotonic Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 21 Myotonic Dystrophy - Pipeline by Valentia Biopharma S.L., H1 2016 22 Myotonic Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 23 Assessment by Monotherapy Products, H1 2016 24 Number of Products by Stage and Target, H1 2016 26 Number of Products by Stage and Mechanism of Action, H1 2016 28 Number of Products by Stage and Route of Administration, H1 2016 30 Number of Products by Stage and Molecule Type, H1 2016 32 Myotonic Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 57 Myotonic Dystrophy - Dormant Projects, H1 2016 58



                                

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