Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy.

Gene Ther · 2025 · PMC12518124 · PMID 40269260

Europe PMC DOI

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a decreasing trendno p-value reported

Despite evidence of transaminitis in their last follow-up laboratory tests, which showed a decreasing trend, cardiac enzyme levels were not elevated.

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