Study Discovers Potential New Target for Treatment of Spinal Muscular Atrophy
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. “Restoring mitochondrial function might be a new treatment strategy for SMA,” said Yongchao Ma, PhD, senior author and Ann Marie and Francis Klocke, MD Research Scholar, Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago. Infants born with SMA are not able to hold up their heads or sit up on their own, and they rarely survive beyond 2 years of age. To read more, click here.
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