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References from Editing the rulebook: building the policy environment for children to access gene therapy. Local targets link to admitted publications; unresolved targets remain external evidence.
10.1038/s41390-026-05270-3
10.1038/s41390-026-05270-3 · External reference
10.1016/j.lfs.2024.122685
10.1016/j.lfs.2024.122685 · 2024 · External reference
10.1016/j.pediatrneurol.2021.09.011
10.1016/j.pediatrneurol.2021.09.011 · 2021 · External reference
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10.1080/20016689.2021.2022353
10.1080/20016689.2021.2022353 · 2022 · External reference
10.1001/jama.2024.11703
10.1001/jama.2024.11703 · 2024 · External reference
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10.1377/hlthaff.2020.00595
10.1377/hlthaff.2020.00595 · 2020 · External reference
10.1038/s41587-023-02096-w
10.1038/s41587-023-02096-w · 2024 · External reference
10.1056/nejmoa2504747
10.1056/nejmoa2504747 · 2025 · External reference
10.1056/nejmoa1813279
10.1056/nejmoa1813279 · 2019 · External reference
10.1186/s13023-026-04388-1
10.1186/s13023-026-04388-1 · 2026 · External reference
10.1016/j.ymthe.2025.09.049
10.1016/j.ymthe.2025.09.049 · 2025 · External reference
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