Paediatrics
Paediatrics

Template-assisted sequence knock-in rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy

Mol Ther. 2025 May 7:S1525-0016(25)00375-2. doi: 10.1016/j.ymthe.2025.05.005. Online ahead of print. ABSTRACT Duchenne muscular dystrophy (DMD) poses challenges in therapy design due to dystrophin’s complex …

Anatomical insights and management strategies for haemodynamically significant pressure-restrictive perimembranous ventricular septal defects: Findings from the French nationwide FRANCISCO cohort

Arch Cardiovasc Dis. 2025 Apr 14:S1875-2136(25)00219-0. doi: 10.1016/j.acvd.2025.02.014. Online ahead of print. ABSTRACT BACKGROUND: Management of haemodynamically significant pressure-restrictive perimembranous ventricular septal defects (pmVSDs) with …