Muscular Dystrophy – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Muscular Dystrophy – Pipeline Review, H1 2017’, provides an overview of the Muscular Dystrophy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Muscular 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 Muscular 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 Muscular Dystrophy

The report reviews pipeline therapeutics for Muscular 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 Muscular Dystrophy therapeutics and enlists all their major and minor projects

The report assesses Muscular 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 Muscular 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 Muscular 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 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

Companies mentioned

Acceleron Pharma Inc

Achelios Therapeutics Inc

AMO Pharma Ltd

aTyr Pharma Inc

Benitec Biopharma Ltd

Bio Blast Pharma Ltd

Biophytis SAS

Corcept Therapeutics Inc

Evotec AG

F. Hoffmann-La Roche Ltd

Fulcrum Therapeutics Inc

Genervon Biopharmaceuticals LLC

Genethon SA

Ionis Pharmaceuticals Inc

Marina Biotech Inc

Medestea Research & Production SpA

Novogen Ltd

Pfizer Inc

Prothelia Inc

SanBio Inc

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics Inc

Strykagen Corp

Takeda Pharmaceutical Company Ltd

Ultragenyx Pharmaceutical Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

Table of Contents 2

Introduction 4

Muscular Dystrophy - Overview 5

Muscular Dystrophy - Therapeutics Development 6

Muscular Dystrophy - Therapeutics Assessment 15

Muscular Dystrophy - Companies Involved in Therapeutics Development 25

Muscular Dystrophy - Drug Profiles 38

Muscular Dystrophy - Dormant Projects 116

Muscular Dystrophy - Discontinued Products 118

Muscular Dystrophy - Product Development Milestones 119

Appendix 131

List of Tables

List of Tables

Number of Products under Development for Muscular Dystrophy, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Companies, H1 2017 (Contd..2), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Target, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017 (Contd..1), H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Muscular Dystrophy – Pipeline by Acceleron Pharma Inc, H1 2017

Muscular Dystrophy – Pipeline by Achelios Therapeutics Inc, H1 2017

Muscular Dystrophy – Pipeline by AMO Pharma Ltd, H1 2017

Muscular Dystrophy – Pipeline by aTyr Pharma Inc, H1 2017

Muscular Dystrophy – Pipeline by Benitec Biopharma Ltd, H1 2017

Muscular Dystrophy – Pipeline by Bio Blast Pharma Ltd, H1 2017

Muscular Dystrophy – Pipeline by Biophytis SAS, H1 2017

Muscular Dystrophy – Pipeline by Corcept Therapeutics Inc, H1 2017

Muscular Dystrophy – Pipeline by Evotec AG, H1 2017

Muscular Dystrophy – Pipeline by F. Hoffmann-La Roche Ltd, H1 2017

Muscular Dystrophy – Pipeline by Fulcrum Therapeutics Inc, H1 2017

Muscular Dystrophy – Pipeline by Genervon Biopharmaceuticals LLC, H1 2017

Muscular Dystrophy – Pipeline by Genethon SA, H1 2017

Muscular Dystrophy – Pipeline by Ionis Pharmaceuticals Inc, H1 2017

Muscular Dystrophy – Pipeline by Marina Biotech Inc, H1 2017

Muscular Dystrophy – Pipeline by Medestea Research & Production SpA, H1 2017

Muscular Dystrophy – Pipeline by Novogen Ltd, H1 2017

Muscular Dystrophy – Pipeline by Pfizer Inc, H1 2017

Muscular Dystrophy – Pipeline by Prothelia Inc, H1 2017

Muscular Dystrophy – Pipeline by SanBio Inc, H1 2017

Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG, H1 2017

Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc, H1 2017

Muscular Dystrophy – Pipeline by Strykagen Corp, H1 2017

Muscular Dystrophy – Pipeline by Takeda Pharmaceutical Company Ltd, H1 2017

Muscular Dystrophy – Pipeline by Ultragenyx Pharmaceutical Inc, H1 2017

Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd, H1 2017

Muscular Dystrophy – Dormant Projects, H1 2017

Muscular Dystrophy – Dormant Projects, H1 2017 (Contd..1), H1 2017

Muscular Dystrophy – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Muscular Dystrophy, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Top 10 Targets, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Top 10 Molecule Types, H1 2017

Number of Products by Stage and Top 10 Molecule Types, H1 2017

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