Historic breakthrough: first in-womb treatment for SMA

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Doctors have achieved a major medical breakthrough by treating a baby for Spinal Muscular Atrophy (SMA) while still in the womb. This innovative approach offers hope for addressing severe genetic disorders.

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Doctors administered Risdiplam to the baby’s mother. The drug helps the body produce SMN protein, which motor neurons need to function. Early treatment was critical to stopping the disease’s progression, which is often fatal without intervention.

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After six weeks of treatment, the baby showed no signs of muscle weakness at birth. SMA usually causes this weakness, so this result marked a major success. The New England Journal of Medicine published the study. It highlights how fetal treatments could address diseases before birth, transforming early-stage care for serious conditions.

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