PhysiologybioRxivPreprint
Combining SMN2 splicing modifiers with HDAC6 inhibition greatly improves muscle function and survival in Spinal Muscular Atrophy
Alexis Osseni, Rasha Slika, Laurent Coudert…·Laurent Schaeffer
This study shows that combining HDAC6 inhibitors with SMN2 splicing modifiers improves muscle function and survival in a SMA mouse model.
DOI 10.1101/2025.01.15.633267