25/02/2025
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A two-and-a-half-year-old girl has become the first person treated for spinal muscular atrophy (SMA) before birth, showing no symptoms of the severe motor-neuron disease.
Her mother took the gene-targeting drug Risdiplam during late pregnancy, a groundbreaking approach approved by the FDA for this case. The child continues to take the drug post-birth.
This breakthrough suggests in-utero treatment could prevent SMA symptoms before they develop, potentially transforming future approaches to genetic disorders.
