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Research Background & Research Ideas: Pompe Disease - Pipeline Review, H1 2016

Press release   •   Jul 19, 2016 06:35 EDT

Global Markets Directs, Pompe Disease - Pipeline Review, H1 2016, provides an overview of the Pompe Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Pompe Disease, 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 Pompe Disease and features dormant and discontinued projects.

Global Markets Directs 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 Directs 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.

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Scope

- The report provides a snapshot of the global therapeutic landscape of PompeDisease

- The report reviews pipeline therapeutics for Pompe Disease 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 Pompe Disease therapeutics and enlists all their major and minor projects

- The report assesses Pompe Disease 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 Pompe Disease

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 Pompe Disease

- 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 its most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Pompe Disease 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

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Table of Content :

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Pompe Disease Overview 8

Therapeutics Development 9

Pipeline Products for Pompe Disease - Overview 9

Pipeline Products for Pompe Disease - Comparative Analysis 10

Pompe Disease - Therapeutics under Development by Companies 11

Pompe Disease - Therapeutics under Investigation by Universities/Institutes 12

Pompe Disease - Pipeline Products Glance 13

Late Stage Products 13

Clinical Stage Products 14

Early Stage Products 15

Pompe Disease - Products under Development by Companies 16

Pompe Disease - Products under Investigation by Universities/Institutes 17

Pompe Disease - Companies Involved in Therapeutics Development 18

Alexion Pharmaceuticals, Inc. 18

Amicus Therapeutics, Inc. 19

Audentes Therapeutics, Inc. 20

BioMarin Pharmaceutical Inc. 21

EpiVax, Inc. 22

Etubics Corporation 23

Genzyme Corporation 24

Greenovation Biotech GmbH 25

Oxyrane Belgium NV 26

Pharming Group N.V. 27

RespireRx Pharmaceuticals Inc. 28

Sarepta Therapeutics, Inc. 29

Pompe Disease - Therapeutics Assessment 30

Assessment by Monotherapy Products 30

Assessment by Combination Products 31

Assessment by Target 32

Assessment by Mechanism of Action 34

Assessment by Route of Administration 36

Assessment by Molecule Type 38

Drug Profiles 40

Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 40

Product Description 40

Mechanism of Action 40

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