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DOMAIN EXPERT ENGAGEMENTS

Number of executives repeatedly engaged by snail & email outreach*

POOL + OUTREACH

1006

Interactions with Platform & by Email *

INTERACTIONS

181

Unique # Participated *

PARTICIPANTS

40

Responses Validated *

VALIDATIONS

10

* Platform engagements are dynamic. Stats are published quarterly.


  •  CODE

  •  PAGES

  •  DATE

  •  PRICE

    $

  •  DATA EXHIBITS

    125


TABLE OF CONTENTS


   Pages :    | $

   Study Reliability and Reporting Limitations.....I-1  
   Disclaimers.....I-2
Data Interpretation & Reporting Level.....I-2
  
   Product Definitions and Scope of Study.....I-3  
   A Prelude.....II-1
Current HD Treatment.....II-1
1$100
   Huntington’s Disease – Quick Facts.....II-21$100
   Market Opportunity.....II-3
Huntington’s Disease Prevalence in Major Region/Countries.....II-3
Competition in the Global HD Therapeutics Market is Non-existent.....II-3
1$100
   Effectiveness of Current HD Management Strategies.....II-41$100
   HD Treatment and Drugs: Brief Description of Disorder, Medication and
  Side-Effects.....II-5
Alternative Treatment Approaches for Huntington’s Disease.....II-5
1$100
   Alternate Approaches and Therapies.....II-6
Off-Label Prescriptions.....II-6
Select Off-Label Drugs Prescribed for Huntington’s Disease: Drug Name & Manufacturer;
  Treatment Class; and Primary Indication.....II-6
1$100
   mHtt Aggregates in HD—Molecular Pathogenesis.....II-71$100
   Therapeutics Focus on Reduction of mHtt Expression.....II-8
Companies Boost R&D Spending for HD Therapeutics.....II-8
Improved Diagnostics Fueling Interest of Companies in HD Research.....II-8
1$100
   Support Organizations Play an Instrumental Role in HD Research and Awareness.....II-9
Public-Private Partnerships: A Shot in the Arm.....II-9
HD Research Attracts 'Big Pharma' Investment.....II-9
1$100
   Pre-Symptomatic Patients to Expand Market Base.....II-10
HDSA Pushes for Improvement in Medicare Funding for Huntington’s Disease.....II-10
1$100
   GINA for Reducing Discrimination Faced by People with HD.....II-111$100
   A Quick Primer.....II-12
Drug Development Pipeline Reveals a Promising Picture.....II-12
Select Drugs and Supplements in HD Research Pipeline (2014): Drug Name, Manufacturer,
  Mode of Action and Current Status.....II-12
1$100
   An Overview of Select Drugs in Late-Stage Clinical Trials.....II-13
SD-809.....II-13
RP103 (Procysbi).....II-13
PBT2.....II-13
1$100
   Huntexil™ (Pridopidine).....II-141$100
   Other Novel Approaches for HD Treatment.....II-15
Cellular Delivery of Neurotropic Agents.....II-15
Cogane.....II-15
1$100
   COPREXA.....II-16
Histone Deacetylases (HDAC).....II-16
Melatonin, Coenzyme Q-Free Radical Scavengers.....II-16
RNAi - RNA Interference.....II-16
1$100
   Triglyceride Oil ‘Triheptanoin’ Shows Promise for HD Therapeutics.....II-17
Reducing mTORC1 Activation through Drug/Diet May be Helpful.....II-17
Better Understanding of Early Neuronal Changes Lead to Targeted Drug
  Development.....II-17
Lund University Researchers Successfully Map Functionality of Hypothalamus in
  HD.....II-17
1$100
   MIB, University of Lisbon and University of Leicester Demonstrate Latest Findings on
  KMO Enzyme.....II-18
Latest TRACK-HD Findings Demonstrate Early Identification of Huntington's Disease
  Progression in Patients.....II-18
Researchers at BUSM, MGH, Columbia University and McLean BTRC Demonstrate Impact of
  Huntington's Disease on Human Brain.....II-18
1$100
   Researchers Demonstrate Significance of TFEB and PGC-1alpha Proteins in Prevention of
  HD.....II-19
Florey Neuroscience Institutes Demonstrates Importance of Cognitive Stimulation in HD
  Patients.....II-19
1$100
   University of Wisconsin-Madison Waisman Center Demonstrates Stem Cell Therapy Potential
  for Treating HD.....II-20
NUI Galway Announces Discovery of Experimental Drugs with Potential for Slowing
  Progress of HD.....II-20
Lawrence Berkeley National Laboratory Designs Compound for Suppressing HD
  Symptoms.....II-20
1$100
   Development of HD Affected Human Brain Cells for Investigative Purposes.....II-21
Device Implant in Brain Exhibits Therapeutic Potential for HD.....II-21
1$100
   Sangamo Biosciences on Path to Discover a Therapy for HD.....II-22
Scientists at University of Rochester Medical Center Demonstrate Use of Coenzyme Q10
  for Reduction of Oxidative Damage in HD Patients.....II-22
1$100
   Introduction.....II-23
Historical Background.....II-23
1$100
   Prognosis.....II-24
Stages of HD.....II-24
1$100
   Symptoms and Signs.....II-25
Physical Symptoms.....II-25
1$100
   Emotional Symptoms.....II-26
Mental Symptoms.....II-26
Genetics of Huntington's Disease.....II-26
1$100
   Inheritance.....II-27
Mechanism.....II-27
1$100
   Functions of HTT.....II-28
Effects of mHTT on Cells.....II-28
Macroscopic Effects of mHTT.....II-28
1$100
   Diagnosis of HD.....II-29
Clinical Diagnosis.....II-29
1$100
   Embryonic Diagnosis.....II-30
Differential Diagnosis.....II-30
Diagnostic Tests for Huntington's Disease.....II-30
Confirmatory Diagnostic Test.....II-30
Pre-symptomatic Diagnostic Test.....II-30
Prenatal Diagnostic Test.....II-30
Treatment Options.....II-30
1$100
   Nutrition and Eating.....II-31
Social Activity.....II-31
Speech Therapy.....II-31
Physical Therapy.....II-31
1$100
   Occupational Therapy.....II-32
Medication.....II-32
Molecular Chaperones.....II-32
2$200
   Auspex Reports Favorable Study Results of SD-809.....II-34
Sangamo and Collaborators to Present ZFP Therapeutics® Data.....II-34
Sangamo Presents Positive Preclinical Data of ZFP Therapeutic®.....II-34
Sangamo BioSciences Acquires Ceregene.....II-34
Prana Completes Reach2HD Trial.....II-34
1$100
   Teva Pharmaceuticals Forms Global R&D Collaboration for HD.....II-35
KineMed, Isis and CHDI Foundation Collaborate for HD Therapeutics.....II-35
CHDI Foundation Continues Collaboration with Medtronic.....II-35
Roche Enters into Alliance with Isis Pharmaceuticals.....II-35
Omeros Receives IND Approval for OMS824 for Huntington’s Disease.....II-35
Teva Pharmaceutical Industries Signs Asset Transfer Agreement with NeuroSearch.....II-35
uniQure and Benitec Biopharma Ink Cross-Licensing Agreement.....II-35
1$100
   Evotec and CHDI Foundation Extend Collaboration.....II-36
Raptor Acquires Exclusive Rights to Cysteamine and Associated Compounds.....II-36
GNS Healthcare Enters into Collaboration with CHDI Foundation.....II-36
Lundbeck Collaborates with CHDI Foundation for HD Research.....II-36
Horizon Discovery Collaborates with Institute of Neurology, London.....II-36
Thomson Reuters and CHDI Foundation Enter into MetaMiner Partnership.....II-36
1$100
   Alnylam Pharmaceuticals, Inc. (US).....II-37
Auspex Pharmaceuticals, Inc. (US).....II-37
Intellect Neurosciences Incorporation (US).....II-37
1$100
   Lundbeck A/S (Denmark).....II-38
Prana Biotechnology Limited (Australia).....II-38
Raptor Pharmaceuticals Corp. (US).....II-38
1$100
   Sangamo Biosciences, Inc. (US).....II-39
Teva Pharmaceutical Industries Ltd (Israel).....II-39
Trophos SA (France).....II-39
1$100
   Valeant Pharmaceuticals International, Inc. (Canada).....II-40
Vertex Pharmaceuticals Incorporated (US).....II-40
1$100
   Table 1: World Recent Past, Current & Future Analysis for Huntington's Disease Therapeutics by Geographic Region - US, Europe, and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....II-411$350
   Table 2: World Historic Review for Huntington's Disease Therapeutics by Geographic Region - US, Europe, and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....II-421$350
   Table 3: World 11-Year Perspective for Huntington’s Disease Therapeutics by Geographic Region - Percentage Breakdown of Dollar Sales for US, Europe, and Rest of World Markets for the Years 2009, 2015 & 2020 (includes corresponding Graph/Chart).....II-431$350
   A. Market Analysis.....III-1
HDSA Brings about Improved Medicare Funding for Huntington’s Disease.....III-1
GINA for Reducing Discrimination Faced by People with HD.....III-1
1$75
   Strategic Corporate Developments.....III-22$150
   Select Players.....III-42$150
   B. Market Analytics.....III-6
Table 4: The US Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-6
1$200
   Table 5: The US Historic Review for Huntington's Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-71$200
   Market Analysis.....III-8
Table 6: European Recent Past, Current & Future Analysis for Huntington's Disease Therapeutics by Geographic Region - France, Germany, UK, Spain and Rest of Europe Markets Independently Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-8
1$200
   Table 7: European Historic Review for Huntington's Disease Therapeutics by Geographic Region - France, Germany, UK, Spain and Rest of Europe Markets Independently Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-91$200
   Table 8: European 11-Year Perspective for Huntington's Disease Therapeutics by Geographic Region - Percentage Breakdown of Dollar Revenues for France, Germany, UK, Spain and Rest of Europe Markets for the Years 2009, 2015 & 2020 (includes corresponding Graph/Chart).....III-101$200
   A. Market Analysis.....III-11
Trophos Sa – A Key Player.....III-11
1$75
   B. Market Analytics.....III-12
Table 9: French Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-12
1$200
   Table 10: French Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-131$200
   A. Market Analysis.....III-14
Strategic Corporate Development.....III-14
B. Market Analytics.....III-14
Table 11: German Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-14
1$200
   Table 12: German Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-151$200
   A. Market Analysis.....III-16
Outlook.....III-16
Table 13: Juvenile HD Incidence Estimates in the UK(includes corresponding Graph/Chart).....III-16

Table 14: Juvenile HD Prevalence Estimates in the UK (2006-2010) (includes corresponding Graph/Chart).....III-16
1$200
   Strategic Corporate Development.....III-17
B. Market Analytics.....III-17
Table 15: The UK Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-17
1$200
   Table 16: The UK Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-181$200
   A. Market Analysis.....III-19
KEY PLAYER - SOM BIOTECH.....III-19
1$75
   B. Market Analytics.....III-20
Table 17: Spanish Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-20
1$200
   Table 18: Spanish Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-211$200
   A. Market Analysis.....III-22
Strategic Corporate Development.....III-22
1$75
   B. Market Analytics.....III-23
Table 19: Rest of Europe Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-23
1$200
   Table 20: Rest of Europe Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-241$200
   A. Market Analysis.....III-25
Strategic Corporate Developments.....III-25
Select Players.....III-25
2$150
   B. Market Analytics.....III-27
Table 21: Rest of World Recent Past, Current & Future Analysis for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2014 through 2020 (includes corresponding Graph/Chart).....III-27
1$200
   Table 22: Rest of World Historic Review for Huntington’s Disease Therapeutics Analyzed with Annual Sales Figures in US$ Million for the Years 2009 through 2013 (includes corresponding Graph/Chart).....III-281$200
    
 
 


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