AAV9-mediated GAA gene therapy following enzyme replacement therapy discontinuation in children with infantile-onset Pompe disease.

EClinicalMedicine · 2026 · PMC13427482 · PMID 42541184

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a decreasing trendno p-value reported

A one-year follow-up of patient 2 revealed a decreasing trend in AST levels, with a slope of −0.192 (95% CI: −0.383 to −0.001).

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an increasing trendno p-value reported

Furthermore, the median CK level across all patients also showed an increasing trend, with a slope of 1.737 (95% CI: 0.481–2.994) ( Fig. 4 A).

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