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

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 thera...

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Veröffentlicht in:JACC. Case reports 2024-08, Vol.29 (16), p.102446
Hauptverfasser: Wang, Wanbing, Zhang, Xinghua, Lyu, Jinhao, Yan, Fei, Lou, Xin
Format: Artikel
Sprache:eng
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Mini-Focus Issue on Congenital Heart Disease
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Zusammenfassung: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.
ISSN:2666-0849
2666-0849
DOI:10.1016/j.jaccas.2024.102446