Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
University of Iowa · University of Pennsylvania · +11 more institutions
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36Topics & keywords
Topics
Keywords
- Medicine
- RPE65
- Visual acuity
- Clinical endpoint
- Randomized controlled trial
- Clinical trial
- Ophthalmology
- Pediatrics
UN Sustainable Development Goals
- Good health and well-being
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