Does the Treatment of Huntington's Disease Represent a Commercial Opportunity?

Table of Contents

• Executive Summary
  • Strategic Considerations
  • Stakeholder Implications
• Introduction
• Overview of Huntington' s Disease
  • The Genetics of Huntington' s Disease
  • Pathophysiology
  • Disease Onset, Symptoms, and Diagnosis
  • Epidemiology and Orphan Disease Status
• Current Treatment Strategies
• Emerging Therapies
  • Disease-Modifying Agents
    • Amarin' s Miraxion
    • Avicena' s HD-02
    • MethylGene and EnVivo' s Phenylbutyrate
    • Neurotrophic Factors: BDNF, GDNF, CNTF
  • Symptomatic Emerging Therapies
    • Prestwick Pharmaceuticals' Xenazine
    • NeuroSearch' s ACR-16
    • Sanofi -Aventis' s Rilutek
    • Medivation' s Dimebon
• Challenges and Opportunities

Tables:

• 1. Inheritance Patterns of Huntington' s Disease
• 2. Effect of CAG Repeats on Huntington' s Disease Development
• 3. Agents in Commercial Development for the Treatment of Huntington' s Disease
• 4. Agents Under Academic Investigation for the Treatment of Huntington' s Disease

Figures:

• 1. Brain Structures Implicated in Huntington' s Disease
• 2. Prevalence of Huntington' s Disease in Select Geographic Regions

Sidebars:

• Novel Approaches to the Treatment of Huntington' s Disease: Stem-Cell Transplantation
• Novel Approaches to the Treatment of Huntington' s Disease: Gene Therapy