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Matched adjusted indirect comparisons estimate treatment effects of gene therapy versus idebenone, suggesting efficacy differences.
To estimate the relative treatment effects of lenadogene nolparvovec gene therapy (LNGT) versus idebenone, we performed two matched adjusted indirect comparisons (MAICs) between idebenone aggregated data from the LEROS study and EAP program, and LNGT individual data from the REFLECT study (bilateral treatment).
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Biousse et al. (2026) studied this question.
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