Duchenne Muscular Dystrophy-Pipeline Review, H1 2017

Duchenne Muscular Dystrophy-Pipeline Review, H1 2017


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Executive Summary

Duchenne Muscular Dystrophy-Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Duchenne Muscular Dystrophy-Pipeline Review, H1 2017, provides an overview of the Duchenne Muscular Dystrophy (Genetic Disorders) pipeline landscape.

Duchenne muscular dystrophy is a condition which causes muscle weakness. DMD is an X-linked disorder. The gene for DMD is present on the X chromosome. It codes for a protein named dystrophin. Dystrophin is essential for providing structural support inside muscle cells. Symptoms usually appear before age 6 and may appear as early as infancy. They include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Duchenne Muscular Dystrophy-Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Duchenne Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 3, 2, 14, 7, 2, 51, 12 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 2, 8 and 1 molecules, respectively.

Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic Disorders).

The pipeline guide reviews pipeline therapeutics for Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic 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 Duchenne Muscular Dystrophy (Genetic 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.



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Table of Contents

Table of Contents 2

Introduction 5

Duchenne Muscular Dystrophy-Overview 6

Duchenne Muscular Dystrophy-Therapeutics Development 7

Duchenne Muscular Dystrophy-Therapeutics Assessment 19

Duchenne Muscular Dystrophy-Companies Involved in Therapeutics Development 29

Duchenne Muscular Dystrophy-Drug Profiles 52

Duchenne Muscular Dystrophy-Dormant Projects 248

Duchenne Muscular Dystrophy-Discontinued Products 251

Duchenne Muscular Dystrophy-Product Development Milestones 252

Appendix 263

List of Figures

Number of Products under Development for Duchenne 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 Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

List of Tables

Number of Products under Development for Duchenne 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 Companies, H1 2017 (Contd..2), 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 Companies, H1 2017 (Contd..3), H1 2017

Products under Development by Companies, H1 2017 (Contd..4), 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

Duchenne Muscular Dystrophy-Pipeline by Acceleron Pharma Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Akashi Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Asklepios BioPharmaceutical Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by BioMarin Pharmaceutical Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Bristol-Myers Squibb Company, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Capricor Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Cardero Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Catabasis Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by CRISPR Therapeutics, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Daiichi Sankyo Company Ltd, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Debiopharm International SA, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Editas Medicine Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017

Duchenne Muscular Dystrophy-Pipeline by FibroGen Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Galapagos NV, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Genethon SA, H1 2017

Duchenne Muscular Dystrophy-Pipeline by GTx Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by GW Pharmaceuticals Plc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Idera Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Italfarmaco SpA, H1 2017

Duchenne Muscular Dystrophy-Pipeline by La Jolla Pharmaceutical Company, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Lexicon Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Marina Biotech Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Merck KGaA, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Milo Biotechnology LLC, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Mitobridge Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Mitochon Pharmaceuticals Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by MyoTherix Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by NicOx SA, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Nippon Shinyaku Co Ltd, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Nobelpharma Co Ltd, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Novartis AG, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Pfizer Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Pluristem Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Prothelia Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by PTC Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by RASRx LLC, H1 2017

Duchenne Muscular Dystrophy-Pipeline by ReveraGen BioPharma Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Santhera Pharmaceuticals Holding AG, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Sarepta Therapeutics Inc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Strykagen Corp, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Summit Therapeutics Plc, H1 2017

Duchenne Muscular Dystrophy-Pipeline by Taiho Pharmaceutical Co Ltd, H1 2017

Duchenne Muscular Dystrophy-Pipeline by WAVE Life Sciences Ltd, H1 2017

Duchenne Muscular Dystrophy-Dormant Projects, H1 2017

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

Duchenne Muscular Dystrophy-Dormant Projects, H1 2017 (Contd..2), H1 2017

Duchenne Muscular Dystrophy-Discontinued Products, H1 2017

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Acceleron Pharma Inc, Akashi Therapeutics Inc, Asklepios BioPharmaceutical Inc, BioMarin Pharmaceutical Inc, Bristol-Myers Squibb Company, Capricor Therapeutics Inc, Cardero Therapeutics Inc, Catabasis Pharmaceuticals Inc, CRISPR Therapeutics, Daiichi Sankyo Company Ltd, Debiopharm International SA, Editas Medicine Inc, Eloxx Pharmaceuticals Ltd, FibroGen Inc, Galapagos NV, Genethon SA, GTx Inc, GW Pharmaceuticals Plc, Idera Pharmaceuticals Inc, Italfarmaco SpA, La Jolla Pharmaceutical Company, Lexicon Pharmaceuticals Inc, Marina Biotech Inc, Merck KGaA, Milo Biotechnology LLC, Mitobridge Inc, Mitochon Pharmaceuticals Inc, MyoTherix Inc, NicOx SA, Nippon Shinyaku Co Ltd, Nobelpharma Co Ltd, Novartis AG, Pfizer Inc, Pluristem Therapeutics Inc, Prothelia Inc, PTC Therapeutics Inc, RASRx LLC, ReveraGen BioPharma Inc, Santhera Pharmaceuticals Holding AG, Sarepta Therapeutics Inc, Strykagen Corp, Summit Therapeutics Plc, Taiho Pharmaceutical Co Ltd, WAVE Life Sciences Ltd

Duchenne Muscular Dystrophy Therapeutic Products under Development, Key Players in Duchenne Muscular Dystrophy Therapeutics, Duchenne Muscular Dystrophy Pipeline Overview, Duchenne Muscular Dystrophy Pipeline, Duchenne Muscular Dystrophy Pipeline Assessment

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