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, Inês B. Martins, Lan Vi Ngoc Nguyen, Chrysanthi‐Maria Moysidou, Noelle Findeisen, Ina‐Maria Rudolph, Christina Bukas, Donatella Cea, Gary Jonathan Bassell, Wilfried Rossoll, Marie Piraud, Sebastian Diecke, 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.
bioRxiv · Aug 24, 2026 · PreprintBioengineering