A viewpoint about Lenadogene nolparvovec failing to meet its primary endpoint even though it permanently corrects the m.11778G>A mutation causative of LHON
Eye (Lond)
.
2025 Aug;39(11):2110-2111.
doi: 10.1038/s41433-025-03856-5.
Epub 2025 May 20.
Authors
Isabella Panfoli
1
,
Silvia Ravera
2
3
Affiliations
1
Dipartmento di Farmacia, Università di Genova, Genova, Italy. isabella.panfoli@unige.it.
2
Dipartimento di Medicina Sperimentale, Università di Genova, Genova, Italy.
3
IRCCS Ospedale Policlinico San Martino, Genova, Italy.
PMID:
40394266
PMCID:
PMC12274447
(available on
2026-08-01
)
DOI:
10.1038/s41433-025-03856-5
No abstract available