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

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

• Mandos LLC • Cyclo Therapeutics • Orphazyme • IntraBio Inc • Synaptogenix • Evox Therapeutics • SOM Biotech • Sarepta Therapeutics/StrideBio • ENDECE • Amicus Therapeutics