We report the novel finding of a widespread reduction in myocardial native T1 values and size of abnormal regions following gene therapy in a pediatric patient with infantile-onset Pompe disease. We emphasize the importance of serial cardiac magnetic resonance in assessing the efficacy of gene therapy and monitoring myocardial alterations in Pompe disease.
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|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405949 | PMC |
| http://dx.doi.org/10.1016/j.jaccas.2024.102446 | DOI Listing |
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