Table of Content

Introduction
Executive Summary
Niemann-Pick Type C Disease: Overview
• Causes
• Mechanism of Action
• Signs and Symptoms
• Diagnosis
• Disease Management
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Late Stage Products (Preregistration)
• Comparative Analysis
Arimoclomol: Orphazyme
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report?..
Mid Stage Products (Phase II)
• Comparative Analysis
IB1001: IntraBio
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report?..
Preclinical stage products
• Comparative Analysis
Research programme: Niemann-Pick disease gene therapies - Sarepta Therapeutics/StrideBio
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report?..
Inactive Products
• Comparative Analysis
Niemann-Pick Type C Disease Key Companies
Niemann-Pick Type C Disease Key Products
Niemann-Pick Type C Disease- Unmet Needs
Niemann-Pick Type C Disease- Market Drivers and Barriers
Niemann-Pick Type C Disease- Future Perspectives and Conclusion
Niemann-Pick Type C Disease Analyst Views
Niemann-Pick Type C Disease Key Companies
Appendix



List of Figures

Figure 1 Total Products for Niemann-Pick Type C Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

List of Tables

Table 1 Total Products for Niemann-Pick Type C Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products