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Chief Healthcare Executive December 21, 2024
Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease
MedPage Today December 22, 2024
— However, FDA approval isn’t expected soon due to quirk in earlier results
Patients with Leber hereditary optic neuropathy (LHON) due to the MT-ND4 gene variant treated with the gene therapy lenadogene nolparvovec in one eye showed a sustained improvement in best-corrected visual acuity (BCVA) in both eyes up to 5 years after treatment, the long-term RESTORE study showed.
Among 55 patients who completed the 5-year follow-up, bilateral vision improvement was similar to what was observed at the 2-year mark, with a mean change in BCVA of -0.4 logMAR (more than +4 lines) for treated eyes and -0.4 logMAR (+4 lines) for eyes treated with sham (difference -0.05, 95% CI -0.15 to 0.04, P=0.27), reported Patrick Yu-Wai-Man, MD, PhD, of...
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