Phenylketonuria Therapeutics - Pipeline Assessment and

Market Forecasts to 2019

On 18thNovember 2014

Summary
The industry analysis specialist, has released its new report, Phenylketonuria Therapeutics Pipeline Assessment and Market Forecasts to 2019. The report is an essential source of
information and analysis on the global Phenylketonuria Therapeutics market. The report
identifies the key trends shaping and driving the global Phenylketonuria Therapeutics market.
The report also provides insights on the prevalent competitive landscape and the emerging
players expected to significantly alter the market positioning of the current market leaders.
Most importantly, the report provides valuable insights on the pipeline products within the
global Phenylketonuria Therapeutics sector. This report is built using data and information
sourced from proprietary databases, primary and secondary research and in-house analysis by
GlobalDatas team of industry experts.

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Scope
The report provides information on the key drivers and challenges of the Phenylketonuria
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Therapeutics market. Its scope includes - Annualized seven key markets (US, France, Germany, Italy, Spain, UK and Japan)
Phenylketonuria Therapeutics market revenues data from 2006 to 2011, forecast for eight years
to 2019.
- Pipeline analysis data providing a split across the different phases, mechanisms of action being
developed and emerging trends by seven key markets. Pipeline candidates fall under major
therapeutic classes.
- Analysis of the current and future competition in the seven key countries Phenylketonuria
Therapeutics market.
- Insightful review of the key industry drivers, restraints and challenges. Each trend is
independently researched to provide a qualitative analysis of its implications.
- Key topics covered include strategic competitor assessment, market characterization, unmet
needs and the implications for the Phenylketonuria Therapeutics market.
- Analysis of key recent licensing and partnership agreements in Phenylketonuria Therapeutics
market

Reasons to buy
The report will enhance your decision making capability. It will allow you to - Develop and design your in-licensing and out-licensing strategies through a review of pipeline
products and technologies and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global
Phenylketonuria Therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies,
market segments and companies likely to impact the global Phenylketonuria Therapeutics
market in future.
- Formulate effective sales and marketing strategies by understanding the competitive
landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective
counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments
that present maximum opportunities for consolidations, investments and strategic
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partnerships.
- Whats the next big thing in the global Phenylketonuria Therapeutics market landscape?
Identify, understand and capitalize.

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Table of Content
2 Phenylketonuria Therapeutics - Introduction
2.1 Overview
2.2 Epidemiology
2.3 Etiology
2.4 Signs and Symptoms
2.5 Diagnosis
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2.6 Treatment
2.7 Management
2.8 Referral Pathway for Phenylketonuria
2.9 GlobalData Pipeline Report Guidance
3 Phenylketonuria Therapeutics - Market Characterization
3.1 Phenylketonuria Therapeutics Market Size (2007-2011) - Global
3.2 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Global
3.3 Phenylketonuria Therapeutics Market Size (2007-2011) - The US
3.4 Phenylketonuria Therapeutics Market Forecast (2011-2019) - The US
3.5 Phenylketonuria Therapeutics Market Size (2008-2011) - The UK
3.6 Phenylketonuria Therapeutics Market Forecast (2011-2019) - The UK
3.7 Phenylketonuria Therapeutics Market Size (2008-2011) - France
3.8 Phenylketonuria Therapeutics Market Forecast (2011-2019) - France
3.9 Phenylketonuria Therapeutics Market Size (2008-2011) - Germany
3.10 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Germany
3.11 Phenylketonuria Therapeutics Market Size (2008-2011) - Italy
3.12 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Italy
3.13 Phenylketonuria Therapeutics Market Size (2008-2011) - Spain
3.14 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Spain
3.15 Phenylketonuria Therapeutics Market Size (2008-2011) - Japan
3.16 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Japan
3.17 Drivers and Barriers for the Phenylketonuria Therapeutics Market
3.17.1 Drivers for the Phenylketonuria Therapeutics Market
3.17.2 Barriers for the Phenylketonuria Therapeutics Market
3.18 Key Events Impacting the Future Market
3.19 Opportunity and Unmet Need
3.19.1 Opportunity and Unmet Need for the Phenylketonuria Therapeutics Market
3.20 Key Takeaway
4 Phenylketonuria Therapeutics Market - Competitive Assessment
4.1 Overview
4.1.1 Strategic Competitive Assessment
4.2 Product Profiles of the Marketed Products in the Phenylketonuria Market
4.2.1 Kuvan (sapropterin dihydrochloride)
4.3 Key Takeaway
5 Phenylketonuria Therapeutics Market - Pipeline Assessment
5.1 Overview
5.2 Strategic Pipeline Assessment
5.2.1 Phenylketonuria Therapeutics - Phase II Pipeline
5.3 Phenylketonuria Pipeline by Mechanism of Action
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5.4 Phenylketonuria Technology Trends Analytical Framework

5.5 Phenylketonuria Therapeutics - Drugs under Clinical Development
5.6 Profile of Drugs under Clinical Development
5.6.1 PEG-PAL
5.7 Key Takeaway
6 Phenylketonuria Therapeutics - Clinical Trials Mapping
6.1 Clinical Trials Mapping by Country (The US, EU5 and Japan)
6.2 Clinical Trials Mapping by Phase
6.3 Clinical Trials Mapping by Trial Status
6.4 Clinical Trials Mapping by Overall Sponsors
6.5 Clinical Trial Mapping by Prominent Sponsors
6.6 Top Companies Participating in Phenylketonuria Therapeutics Clinical Trials
7 Phenylketonuria Therapeutics - Strategic Assessment
7.1 Future Market Competition Scenario
7.2 Key Takeaway
8 Global Phenylketonuria Therapeutics - Future Players in The Market
8.1 Introduction
8.2 Company Profile
8.2.1 BioMarin Pharmaceuticals Inc.
9 Phenylketonuria Therapeutics - Licensing and Partnership Deals
10 Phenylketonuria Therapeutics - Appendix
10.1 Acronyms
10.2 Bibliography
10.3 Research Methodology
10.3.1 Coverage
10.3.2 Secondary Research
10.3.3 Primary Research
10.3.4 Forecasting
10.3.5 Expert Panel Validation
10.4 Contact Us
10.5 Disclaimer
List Of Tables
Table 1: Biochemical Basis for Phenylketonuria
Table 2: Historical Prevalence for Phenylketonuria for Seven Major Markets, 2006-2011
Table 3: Forecasted Prevalence for Phenylketonuria for Seven Major Markets, 2011-2019
Table 4: Characteristics of Therapeutics for BH4 responsive phenylketonuria patients
Table 5: Phenylketonuria Therapeutics Market, Seven Major Markets, Revenue ($m), 2007-2011
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Table 6: Phenylketonuria Therapeutics Market, Seven Major Markets, Market Forecast ($m),
2011-2019
Table 7: Phenylketonuria Therapeutics Market, The US, Revenue ($m), 2007-2011
Table 8: Phenylketonuria Therapeutics Market, The US, Market Forecast ($m), 2011-2019
Table 9: Phenylketonuria Therapeutics Market, The UK, Revenue ($m), 2008-2011
Table 10: Phenylketonuria Therapeutics Market, The UK, Market Forecast ($m), 2011-2019
Table 11: Phenylketonuria Therapeutics Market, France, Revenue ($m), 2008-2011
Table 12: Phenylketonuria Therapeutics Market, France, Market Forecast ($m), 2011-2019
Table 13: Phenylketonuria Therapeutics Market,Germany, Revenue ($m), 2008-2011
Table 14: Phenylketonuria Therapeutics Market, Germany, Market Forecast ($m), 2011-2019
Table 15: Phenylketonuria Therapeutics Market, Italy, Revenue ($m), 2008-2011
Table 16: Phenylketonuria Therapeutics Market, Italy, Market Forecast ($m), 2011-2019
Table 17: Phenylketonuria Therapeutics Market, Spain, Revenue ($m), 2008-2011
Table 18: Phenylketonuria Therapeutics Market, Spain, Market Forecast ($m), 2011-2019
Table 19: Phenylketonuria Therapeutics Market, Japan, Revenue ($m), 2008-2011
Table 20: Phenylketonuria Therapeutics Market, Japan, Market Forecast ($m), 2011-2019
Table 21: Phenylketonuria - Phase II Pipeline
Table 22: Phenylketonuria Therapeutics - Promising Drugs Under Clinical Development, 2012
Table 23: Phenylketonuria Therapeutics - Clinical Trials by Country, 2012
Table 24: Phenylketonuria Therapeutics - Clinical Trials by Phase, 2012
Table 25: Phenylketonuria Therapeutics - Clinical Trials by Status, 2012
Table 26: Phenylketonuria Therapeutics - Clinical Trials by Overall Sponsors, 2012
Table 27: Phenylketonuria Therapeutics - Clinical trials by Prominent Sponsors, 2012
Table 28: Phenylketonuria Therapeutics - Clinical Trials by Top Companies Participating in
Therapeutics Clinical Trials, 2012
Table 29: Phenylketonuria Therapeutics, Clinical Pipeline by Company, 2012
Table 30: BioMarin Pharmaceuticals Inc. - Promising Molecules in the Pipeline for
Phenylketonuria, 2012
Table 31: PhenylketonuriaTherapeutics, Global, Deals ($m), 2012
List Of Figures
Figure 1: Factors Modulating Phenylketonuria
Figure 2: Patient Share of Phenylketonuria in the Seven Major Markets, 2011
Figure 3: Mechanism of Formation of Phenylketonuria
Figure 4: The Basic Structure of the Human PAH Gene
Figure 5: Allelic Mutations Associated with Phenylketonuria
Figure 6: Treatment Algorithm for Phenylketonuria
Figure 7: Referral Pathway for Phenylketonuria
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Figure 8: Phenylketonuria Therapeutics Market, Seven Major Markets, Revenue ($m), 20072011
Figure 9: Phenylketonuria Therapeutics Market, Market Shares (%), 2011
Figure 10: Phenylketonuria Therapeutics Market, Seven Major Markets, Forecast ($m), 20112019
Figure 11: Phenylketonuria Therapeutics, Market Share (%), 2019
Figure 12: Phenylketonuria Therapeutics Market, The US, Revenue ($m), 2007-2011
Figure 13: Phenylketonuria Therapeutics Market, The US, Market Forecast ($m), 2011-2019
Figure 14: Phenylketonuria Therapeutics Market, The UK, Revenue ($m), 2008-2011
Figure 15: Phenylketonuria Therapeutics Market, The UK, Market Forecast ($m), 2011-2019
Figure 16: Phenylketonuria Therapeutics Market, France, Revenue ($m), 2008-2011
Figure 17: Phenylketonuria Therapeutics Market, France, Market Forecast ($m), 2011-2019
Figure 18: Phenylketonuria Therapeutics Market, Germany, Revenue ($m), 2008-2011
Figure 19: Phenylketonuria Therapeutics Market, Germany, Market Forecast ($m), 2011-2019
Figure 20: Phenylketonuria Therapeutics Market, Italy, Revenue ($m), 2008-2011
Figure 21: Phenylketonuria Therapeutics Market, Italy, Market Forecast ($m), 2011-2019
Figure 22: Phenylketonuria Therapeutics Market, Spain, Revenue ($m), 2008-2011
Figure 23: Phenylketonuria Therapeutics Market, Spain, Market Forecast ($m), 2011-2019
Figure 24: Phenylketonuria Therapeutics Market, Japan, Revenue ($m), 2008-2011
Figure 25: Phenylketonuria Therapeutics Market, Japan, Market Forecast ($m), 2011-2019
Figure 26: Phenylketonuria Therapeutics, Key Events Impacting Future Market, 2012
Figure 27: Opportunity and Unmet Need in the Phenylketonuria Therapeutics Market, 2012
Figure 28: Phenylketonuria Market - Strategic Competitor Assessment, 2012
Figure 29: Mechanism of Action of Kuvan
Figure 30: Technology Trends Analytical Framework of the Phenylketonuria Pipeline Description, 2012
Figure 31: Phenylketonuria Therapeutics - Clinical Trials by Country, 2012
Figure 32: Phenylketonuria Therapeutics - Clinical Trials by Phase, 2012
Figure 33: Phenylketonuria Therapeutics - Clinical Trials by Status, 2012
Figure 34: Phenylketonuria Therapeutics - Clinical Trials by Overall Sponsors, 2012
Figure 35: Phenylketonuria Therapeutics - Clinical Trials by Prominent Sponsors, 2012
Figure 36: Phenylketonuria Therapeutics Clinical Trials - Company Sponsors by Phase, 2012
Figure 37: Phenylketonuria Therapeutics, Strategic Assessment, 2012
Figure 38: GlobalData market Forecasting Model, 2012

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