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Why this trial matters : First effective gene therapy for EB Meaningfully improves quality of life and patient outcomes Drug Approvals as a Signal: 2025 Showed Real Late-Stage Productivity The FDAs 2025 novel drug approvals, alongside pivotal trialdriven cancer approvals, point to real late-stage productivity rather than incremental gains

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The resulting interplay between receptor-mediated targeting and redox-triggered release sustains localized immune modulation and oxidative balance in the regenerating tissue with permission ) [23]