Barely Significant
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Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy.

Mol Ther Methods Clin Dev · 2024 · PMC11866129 · PMID 40017665

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a clear trendno p-value reported
Elevated troponin levels in patients with SMA from birth without further abnormalities upon cardiac evaluation are emerging as a clear trend with unclear clinical significance. 29 , 30 Conclusion We have presented 2 cases of liver injury with moderate to severe elevations in liver enzymes, without evidence of liver failure, in infants with SMA following treatment with OA at less than 4 weeks old.

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