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

LHON

Eye (Lond). 2025 May 20. doi: 10.1038/s41433-025-03856-5. Online ahead of print.

NO ABSTRACT

PMID:40394266 | DOI:10.1038/s41433-025-03856-5