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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Global Clinical Trials Review, H1, 2014

Summary

GlobalData's clinical trial report, “Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Global Clinical Trials Review, H1, 2014" provides data on the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) clinical trial scenario. This report provides elemental information and data relating to the clinical trials on Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome). 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome). 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- Listings of discontinued trials (suspended, withdrawn and terminated)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 Contents

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Global Clinical Trials Review, H1, 2014
Table of Contents
Table of Contents2
List of Tables 4
List of Figures 5
Introduction 6
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 6
Report Guidance 6
Clinical Trials by Region 7
Clinical Trials and Average Enrollment by Country 8
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 Central and South America 12
Clinical Trials by G7 Countries: Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials 13
Clinical Trials by Phase in G7 Countries 14
Clinical Trials in G7 Countries by Trial Status 15
Clinical Trials by E7 Countries: Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials 16
Clinical Trials by Phase 17
In Progress Trials by Phase 18
Clinical Trials by Trial Status 19
Clinical Trials by End Point Status 20
Unaccomplished Trials of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 21
Subjects Recruited Over a Period of Time 22
Clinical Trials by Sponsor Type 23
Prominent Sponsors 24
Top Companies Participating in Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials 25
Prominent Drugs 26
Clinical Trial Profiles 27
Clinical Trial Overview of Top Companies 27
Shire Plc 27
Clinical Trial Overview of Shire Plc 27
UniQure NV 28
Clinical Trial Overview of UniQure NV 28
Lysogene 29
Clinical Trial Overview of Lysogene 29
Synageva BioPharma Corp. 30
Clinical Trial Overview of Synageva BioPharma Corp. 30
Cytomedix, Inc. 31
Clinical Trial Overview of Cytomedix, Inc. 31
BioMarin Pharmaceutical Inc. 32
Clinical Trial Overview of BioMarin Pharmaceutical Inc. 32
Clinical Trial Overview of Top Institutes / Government 33
Academic Medical Center 33
Clinical Trial Overview of Academic Medical Center 33
Masonic Cancer Center 34
Clinical Trial Overview of Masonic Cancer Center 34
The University of Manchester 35
Clinical Trial Overview of The University of Manchester 35
The University of Chicago 36
Clinical Trial Overview of The University of Chicago 36
Hospices Civils de Lyon 37
Clinical Trial Overview of Hospices Civils de Lyon 37
University of Louisville 38
Clinical Trial Overview of University of Louisville 38
University of Minnesota 39
Clinical Trial Overview of University of Minnesota 39
Five Key Clinical Profiles 40
Appendix 62
Abbreviations 62
Definitions 62
Research Methodology 63
Secondary Research 63
About GlobalData 64
Contact Us 64
Disclaimer 64
Source 64

List of Tables
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Region, 2014* 7
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2014* 8
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2014* 9
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Europe, Top Five Countries, 2014* 10
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, North America, Top Countries, 2014* 11
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Central and South America, Top Countries, 2014* 12
Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials, G7 Countries (%), 2014* 13
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, G7 Countries, Clinical Trials by Phase, 2014* 14
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2014* 15
Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials, E7 Countries (%), 2014* 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Phase, 2014* 17
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials In Progress by Phase 2014* 18
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Trial Status, 2014* 19
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, by End Point Status, 2014* 20
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Suspended Clinical Trials, 2014* 21
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2009-2013 22
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Market, Global, Clinical Trials by Sponsor Type, 2014* 23
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Key Sponsors, 2014* 24
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2014* 25
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2014* 26
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Shire Plc, 2014* 27
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by UniQure NV, 2014* 28
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Lysogene, 2014* 29
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Synageva BioPharma Corp., 2014* 30
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Cytomedix, Inc., 2014* 31
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by BioMarin Pharmaceutical Inc., 2014* 32
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Academic Medical Center, 2014* 33
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Masonic Cancer Center, 2014* 34
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by The University of Manchester, 2014* 35
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by The University of Chicago, 2014* 36
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by Hospices Civils de Lyon, 2014* 37
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by University of Louisville, 2014* 38
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials Market, Global, Clinical Trials by University of Minnesota, 2014* 39

List of Figures
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Region (%), 2014* 7
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2014* 8
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2014* 9
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Europe, Top Five Countries (%), 2014* 10
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Central and South America, Top Countries (%), 2014* 12
Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials, G7 Countries (%), 2014* 13
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, G7 Countries, Clinical Trials by Phase, 2014* 14
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2014* 15
Proportion of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) to Metabolic Disorders Clinical Trials, E7 Countries (%), 2014* 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Phase (%), 2014* 17
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials In Progress by Phase, 2014* 18
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics, Global, Clinical Trials by Trial Status, 2014* 19
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, by End Point Status, 2014* 20
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2009-2013 22
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Market, Global, Clinical Trials by Sponsor Type (%), 2014* 23
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Key Sponsors, 2014* 24
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2014* 25
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2014* 26
GlobalData Methodology 63

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