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Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation

Carelli, Valerio, Nancy Newman, Patrick Yu-Wai-Man, Valérie Biousse, Mark Moster, Prem Subramanian, Catherine Vignal-Clermont, et al. 2023. “Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the M.11778G>A MT-ND4 Mutation”. Ophthalmol Ther 12 (1): 401-29.