Open access
Aug 2026
Givinostat enhances antisense oligonucleotide efficacy in the mdx52 model of Duchenne muscular dystrophy
Givinostat significantly improved exon skipping and dystrophin restoration in human DMD myoblasts carrying an exon 52 deletion, and combining givinostat with ASOs to enhance dystrophin restoration and muscle preservation in DMD.
Xaysongkhame Phongsavanh, C. Gastaldi, Astrid Mottais et al.
· Molecular Therapy: Nucleic A... · 0 citations