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Summary
Global Markets Direct’s, Pompe Disease - Pipeline Review, H1 2012, provides an
overview of the Pompe Disease therapeutic pipeline. This report provides
information on the therapeutic development for Pompe Disease, complete with
latest updates, and special features on late-stage and discontinued projects. It also
reviews key players involved in the therapeutic development for Pompe Disease.
Pompe Disease - Pipeline Review, H1 2012 is built using data and information
sourced from Global Markets Direct’s proprietary databases, Company/University
websites, SEC filings, investor presentations and featured press releases from
company/university sites and industry-specific third party sources, put together by
Global Markets Direct’s team.
Note*: Certain sections in the report may be removed or altered based on the
availability and relevance of data for the indicated disease.
Scope
- A snapshot of the global therapeutic scenario for Pompe Disease.
- A review of the Pompe Disease products under development by companies and
universities/research institutes based on information derived from company and
industry-specific sources.
- Coverage of products based on various stages of development ranging from

2. discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined
therapeutics.
Table of contents:
List of Tables 3
List of Figures 3
Introduction 4
Global Markets Direct Report Coverage 4
Pompe Disease Overview 5
Therapeutics Development 6
An Overview of Pipeline Products for Pompe Disease 6
Pompe Disease Therapeutics under Development by Companies 8
Pompe Disease Therapeutics under Investigation by Universities/Institutes 9
Mid Clinical Stage Products 10
Comparative Analysis 10
Pre-Clinical Stage Products 11
Comparative Analysis 11
Pompe Disease Therapeutics – Products under Development by Companies 12
Pompe Disease Therapeutics – Products under Investigation by
Universities/Institutes 13
Companies Involved in Pompe Disease Therapeutics Development 14
Genzyme Corporation 14
BioMarin Pharmaceutical Inc. 15
Pompe Disease – Therapeutics Assessment 16
Assessment by Monotherapy Products 16
Assessment by Route of Administration 17
Assessment by Molecule Type 19
Drug Profiles 21
BMN-701 - Drug Profile 21
Product Description 21
Mechanism of Action 21
R&D Progress 21
rAAV1-CMV-GAA - Drug Profile 23
Product Description 23

3. Mechanism of Action 23
R&D Progress 23
Neo-GAA - Drug Profile 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
Pompe Disease Therapeutics – Drug Profile Updates 25
Pompe Disease Therapeutics – Discontinued Products 26
Pompe Disease Therapeutics - Dormant Products 27
Appendix 28
Methodology 28
Coverage 28
Secondary Research 28
Primary Research 28
List of Figures
List of Tables are also include.
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