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Neuron 92 , 493504 (2016)

Why this trial matters: First global Phase III success in a disease with no approved targeted therapy Could change management of systemic symptoms and glandular dysfunction Gene Therapy Phase III in Epidermolysis Bullosa Shows Healing Benefit A Phase III gene therapy trial for epidermolysis bullosa (EB) demonstrated significantly better wound healing, less pain, and reduced itching compared with standard care, and the therapy earned FDA approval in 2025 a major advance for this previously untreatable genetic skin disorder