This document summarizes a report on clinical trials for Pompe disease from January 2013. It provides an overview of the number and location of trials worldwide in the first half of 2013. The US, Germany, and China had the most trials. The trials were mostly in late phases and investigated drugs from companies like Genzyme and Sanofi. The report analyzed trial trends over time and provided profiles of top trial sponsors.

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Pompe Disease Global Clinical Trials Review, H1, 2013
Published on January 2013
Report Summary
Pompe Disease Global Clinical Trials Review, H1, 2013
Summary
GlobalData's clinical trial report, 'Pompe Disease Global Clinical Trials Review, H1, 2013" provides data on the Pompe Disease
clinical trial scenario. This report provides elemental information and data relating to the clinical trials on Pompe Disease. It includes
an overview of the trial numbers and their recruitment status as per the site of trial conduction across the globe. The databook offers a
preliminary coverage of disease clinical trials by their phase, trial status, prominence of the sponsors and also provides briefing
pertaining to the number of trials for the key drugs for treating Pompe Disease. This report is built using data and information sourced
from proprietary databases, primary and secondary research and in-house analysis by GlobalData's team of industry experts.
Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated
disease.
Scope
- Data on the number of clinical trials conducted in North America, South and Central America, Europe, Middle-East and Africa and
Asia-pacific and top five national contributions in each, along with the clinical trial scenario in BRIC nations
- Clinical trial (complete and in progress) data by phase, trial status, subjects recruited and sponsor type
- Listings of discontinued trials (suspended, withdrawn and terminated)
Reasons to buy
- Understand the dynamics of a particular indication in a condensed manner
- Abridged view of the performance of the trials in terms of their status, recruitment, location, sponsor type and many more
- Obtain discontinued trial listing for trials across the globe
- Espy the commercial landscape of the major Universities / Institutes / Hospitals or Companies
Table of Content
Table of Contents
Table of CContents 2
List of Tables 3
List of Figures 4
Introduction 5
Pompe Disease 5
Report Guidance 5
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Clinical Trials by Region 6
Clinical Trials by Country 7
Top Countries Contributing to Clinical Trials in Asia-Pacific 9
Top Five Countries Contributing to Clinical Trials in Europe 10
Top Countries Contributing to Clinical Trials in North America 11
Top Countries Contributing to Clinical Trials in Middle East and Africa 12
Top Countries Contributing to Clinical Trials in Central and South America 13
Clinical Trials by BRIC Nations 14
Clinical Trials by G7 Nations 15
Clinical Trials in G7 Nations by Trial Status 16
Clinical Trials by E7 Nations 17
Clinical Trials in E7 Nations by Trial Status 18
Clinical Trials by Phase 19
In Progress Trials by Phase 20
Clinical Trials by Trial Status 21
Unaccomplished Trials of Pompe Disease 22
Subjects Recruited Over a Period of Time 23
Prominent Sponsors 24
Top Companies Participating in Pompe Disease Therapeutics Clinical Trials 26
Prominent Drug Comparison 28
Clinical Trial Profiles 29
Clinical Trial Overview of Top Companies 29
Genzyme Corporation 29
Clinical Trial Overview of Genzyme Corporation 29
Sanofi 31
Clinical Trial Overview of Sanofi 31
Amicus Therapeutics, Inc. 32
Clinical Trial Overview of Amicus Therapeutics, Inc. 32
BioMarin Pharmaceutical Inc. 33
Clinical Trial Overview of BioMarin Pharmaceutical Inc. 33
O & O Alpan, LLC 34
Clinical Trial Overview of O & O Alpan, LLC 34
Children's Healthcare of Atlanta Inc. 35
Clinical Trial Overview of Children's Healthcare of Atlanta Inc. 35
Clinical Trial Overview of Top Universities / Institutes / Hospitals 36
University of Florida 36
Clinical Trial Overview of University of Florida 36
Duke University 37
Clinical Trial Overview of Duke University 37
National Taiwan University Hospital 38
Clinical Trial Overview of National Taiwan University Hospital 38
Hospital Vall d'Hebron 39
Clinical Trial Overview of Hospital Vall d'Hebron 39
Erasmus University Medical Center 40
Clinical Trial Overview of Erasmus University Medical Center 40
University of Rostock 41
Clinical Trial Overview of University of Rostock 41
Erasmus MC 42
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Clinical Trial Overview of Erasmus MC 42
Rambam Health Care Campus 43
Clinical Trial Overview of Rambam Health Care Campus 43
Centre Hospitalier Universitaire de Nice 44
Clinical Trial Overview of Centre Hospitalier Universitaire de Nice 44
Five Key Clinical Profiles 45
Appendix 75
Abbreviations 75
Definitions 75
Research Methodology 76
Secondary Research 76
About GlobalData 77
Contact Us 77
Disclaimer 77
Source 78
List of Tables
Pompe Disease Therapeutics Market, Global, Clinical Trials by Zone, 2013* 6
Pompe Disease Therapeutics Market, Global, Clinical Trials by Top Countries, 2013* 7
Pompe Disease Therapeutics Market, Global, Clinical Trials In Progress by Top Countries, 2013* 8
Pompe Disease Therapeutics Clinical Trials, Asia-Pacific, Top Countries, 2013* 9
Pompe Disease Therapeutics Clinical Trials, Europe, Top Five Countries, 2013* 10
Pompe Disease Therapeutics Clinical Trials, North America, Top Countries, 2013* 11
Pompe Disease Therapeutics Clinical Trials, Middle East and Africa, Top Countries, 2013* 12
Pompe Disease Therapeutics Clinical Trials, Central and South America, Top Countries, 2013* 13
Pompe Disease Therapeutics Clinical Trials, BRIC Countries, 2013* 14
Pompe Disease Therapeutics Clinical Trials, G7 Countries, 2013* 15
Pompe Disease Therapeutics Market, G7 Countries, Clinical Trials by Trial Status, 2013* 16
Pompe Disease Therapeutics Clinical Trials, E7 Countries, 2013* 17
Pompe Disease Therapeutics Market, E7 Countries, Clinical Trials by Trial Status, 2013* 18
Pompe Disease Therapeutics Market, Global, Clinical Trials by Phase, 2013* 19
Pompe Disease Therapeutics Market, Global, Clinical Trials In Progress by Phase 2013* 20
Pompe Disease Therapeutics Market, Global, Clinical Trials by Trial Status, 2013* 21
Pompe Disease Therapeutics Market, Global, Terminated Clinical Trials, 2013* 22
Pompe Disease Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2007-2012 23
Pompe Disease Therapeutics Clinical Trials, Global, Key Sponsors, 2013* 25
Pompe Disease Therapeutics Clinical Trials, Global, Top Companies by Phase, 2013* 27
Pompe Disease Therapeutics Clinical Trials, Global, Clinical Trials by Prominent Drugs, 2013* 28
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Genzyme Corporation, 2013* 29
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Sanofi, 2013* 31
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Amicus Therapeutics, Inc., 2013* 32
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by BioMarin Pharmaceutical Inc., 2013* 33
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by O & O Alpan, LLC, 2013* 34
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Children's Healthcare of Atlanta Inc., 2013* 35
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Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by University of Florida, 2013* 36
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Duke University, 2013* 37
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by National Taiwan University Hospital, 2013* 38
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Hospital Vall d'Hebron, 2013* 39
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Erasmus University Medical Center, 2013* 40
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by University of Rostock, 2013* 41
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Erasmus MC, 2013* 42
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Rambam Health Care Campus, 2013* 43
Pompe Disease Therapeutics Clinical Trials Market, Global, Clinical Trials by Centre Hospitalier Universitaire de Nice, 2013* 44
List of Figures
Pompe Disease Therapeutics Market, Global, Clinical Trials by Zone (%), 2013* 6
Pompe Disease Therapeutics Market, Global, Clinical Trials by Top Countries, 2013* 7
Pompe Disease Therapeutics Market, Global, Clinical Trials In Progress by Top Countries, 2013* 8
Pompe Disease Therapeutics Clinical Trials, Asia-Pacific, Top Countries (%), 2013* 9
Pompe Disease Therapeutics Clinical Trials, Europe, Top Five Countries (%), 2013* 10
Pompe Disease Therapeutics Clinical Trials, North America, Top Countries (%), 2013* 11
Pompe Disease Therapeutics Clinical Trials, Middle East and Africa, Top Countries (%), 2013* 12
Pompe Disease Therapeutics Clinical Trials, BRIC Countries (%), 2013* 14
Pompe Disease Therapeutics Clinical Trials, G7 Countries (%), 2013* 15
Pompe Disease Therapeutics Market, G7 Countries, Clinical Trials by Trial Status, 2013* 16
Pompe Disease Therapeutics Clinical Trials, E7 Countries (%), 2013* 17
Pompe Disease Therapeutics Market, Global, Clinical Trials by Phase (%), 2013* 19
Pompe Disease Therapeutics Market, Global, Clinical Trials In Progress by Phase, 2013* 20
Pompe Disease Therapeutics Market, Global, Clinical Trials by Trial Status, 2013* 21
Pompe Disease Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2007-2012 23
Pompe Disease Therapeutics Clinical Trials, Global, Key Sponsors, 2013* 24
Pompe Disease Therapeutics Clinical Trials, Global, Top Companies by Phase, 2013* 26
Pompe Disease Therapeutics Clinical Trials, Global, Clinical Trials by Prominent Drugs, 2013* 28
GlobalData Methodology 76
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