Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.
Yu-Wai-Man Patrick, Newman Nancy J, Biousse ValΓ©rie, Carelli Valerio, Moster Mark L, Vignal-Clermont Catherine et al.
Gene therapy for LHON (MT-ND4 variant) showed sustained bilateral visual improvement and good safety up to five years, offering promising long-term benefit for patients.