BioengineeringbioRxivPreprint
A scalable human neuromuscular organoid platform enables lineage-specific analysis of drug responses in spinal muscular atrophy.
Ines Lahmann, Angélica García-Pérez, Ismail Amr El‐Shimy…·Mina Gouti
This study develops a scalable neuromuscular organoid platform to analyze drug responses in spinal muscular atrophy, showing distinct muscle and neuronal responses to SMN2 splicing modulators.