In-Utero Treatment Shows Promise for Spinal Muscular Atrophy
2025-02-20
A two-and-a-half-year-old girl is thriving, showing no signs of spinal muscular atrophy (SMA), a devastating genetic disease, after receiving in-utero treatment with the drug Risdiplam. This groundbreaking study, published in the New England Journal of Medicine, marks a significant advancement. The drug, administered to the mother during late pregnancy and continued after birth, boosts production of the SMN protein crucial for motor neuron survival. This approach offers a potential game-changer for SMA, a leading genetic cause of infant mortality, as previous treatments began only after birth.