Efficacy of lenadogene nolparvovec gene therapy versus idebenone in Leber hereditary optic neuropathy due to the m.11778G>A MT-ND4 variant: two matching adjusted indirect comparisons
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Background: Lenadogene nolparvovec is an intravitreal gene therapy for patients with Leber hereditary optic neuropathy (LHON) carrying the m.11778G>A MT-ND4 variant. Idebenone, a synthetic coenzyme Q10 analogue, is the only appro... ...