Potential new target for treatment of spinal muscular atrophy discovered

For the first time, scientists found that in spinal muscular atrophy (SMA), the affected nerve cells that control muscle movement, or motor neurons, have defects in their mitochondria, which generate energy used by the cell. Impaired mitochondrial function and structure in motor neurons were discovered before symptoms occurred, suggesting a role in disease development. These findings, published in Human Molecular Genetics, point to new possibilities for targeted therapy for SMA.