Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease
Ophthalmology > > Ophthalmology-- However, FDA approval isn't anticipated quickly due to quirk in earlier outcomes
by Randy Dotinga, Contributing Writer, MedPage Today December 22, 2024
Clients with Leber genetic optic neuropathy (LHON) due to the MT-ND4 gene alternative treated with the gene treatment lenadogene nolparvovec in one eye revealed a continual enhancement in best-corrected visual skill (BCVA) in both eyes approximately 5 years after treatment, the long-lasting RESTORE research study revealed.
Amongst 55 clients who finished the 5-year follow-up, bilateral vision enhancement resembled what was o...