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A viewpoint about Lenadogene nolparvovec failing to meet its primary endpoint even though it permanently corrects the m.11778G>A mutation causative of LHON

Crossref DOI link: https://doi.org/10.1038/s41433-025-03856-5

Published Online: 2025-05-20

Published Print: 2025-08

Update policy: https://doi.org/10.1007/springer_crossmark_policy

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