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Spinal muscular atrophy
March 4 , 2025
33 days
201
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- A two-and-a-half-year-old girl has shown no signs of a genetic disorder — known as spinal muscular atrophy (SMA).
- This girl was becoming the first person in the world to be treated for the disease while in the womb.
- The girl’s mother began taking the gene-targeting drug during late pregnancy, and the child continues to take it.
- SMA is a debilitating genetic condition which affects motor neurons that control movement, and leads to progressive muscle weakening.
- About one in every 10,000 births have some form of the condition.
- This was making it a leading genetic cause of death in infants and children.
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