Table of Contents List of Tables List of Figures Introduction Global Markets Direct Report Coverage Usher Syndrome - Overview Usher Syndrome - Therapeutics Development Pipeline Overview Pipeline by Companies Pipeline by Universities/Institutes Products under Development by Companies Products under Development by Universities/Institutes Usher Syndrome - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Usher Syndrome - Companies Involved in Therapeutics Development Amgen Inc Editas Medicine Inc ProQR Therapeutics NV Sanofi Usher Syndrome - Drug Profiles Gene Therapy for Usher Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate MYO7A for Usher Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress NT-501 - Drug Profile Product Description Mechanism Of Action R&D Progress QRX-411 - Drug Profile Product Description Mechanism Of Action R&D Progress QRX-421 - Drug Profile Product Description Mechanism Of Action R&D Progress SAR-421869 - Drug Profile Product Description Mechanism Of Action R&D Progress Usher Syndrome - Dormant Projects Usher Syndrome - Product Development Milestones Featured News & Press Releases May 01, 2017: ProQR to Present Data on QRX-411 at ARVO May 01, 2017: ProQR to Present Data on QRX-421 at ARVO Sep 18, 2012: Neurotech Pharmaceuticals Provides Update On NT-501 Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer

List of Tables Number of Products under Development for Usher Syndrome, H1 2017 Number of Products under Development by Companies, H1 2017 Number of Products under Development by Universities/Institutes, H1 2017 Products under Development by Companies, H1 2017 Products under Development by Universities/Institutes, H1 2017 Number of Products by Stage and Target, H1 2017 Number of Products by Stage and Mechanism of Action, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Usher Syndrome - Pipeline by Amgen Inc, H1 2017 Usher Syndrome - Pipeline by Editas Medicine Inc, H1 2017 Usher Syndrome - Pipeline by ProQR Therapeutics NV, H1 2017 Usher Syndrome - Pipeline by Sanofi, H1 2017 Usher Syndrome - Dormant Projects, H1 2017 List of Figures Number of Products under Development for Usher Syndrome, H1 2017 Number of Products under Development by Companies, H1 2017 Number of Products by Targets, H1 2017 Number of Products by Stage and Targets, H1 2017 Number of Products by Mechanism of Actions, H1 2017 Number of Products by Stage and Mechanism of Actions, H1 2017 Number of Products by Routes of Administration, H1 2017 Number of Products by Stage and Routes of Administration, H1 2017 Number of Products by Molecule Types, H1 2017 Number of Products by Stage and Molecule Types, H1 2017

Amgen Inc Editas Medicine Inc ProQR Therapeutics NV Sanofi