Assessing Gene Therapy Efficacy in Infantile-Onset Pompe Disease: Myocardial Native T1 Values of CMR.

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.