Daijiworld Media Network – New Delhi

New Delhi, Feb 22: In a groundbreaking medical achievement, doctors have successfully treated a life-threatening motor neuron disease in a baby while still in the womb. The case, published in The New England Journal of Medicine, marks the first-ever prenatal treatment for Spinal Muscular Atrophy (SMA)—a rare genetic disorder that weakens muscles and limits movement.

The unborn child was diagnosed with SMA type 1, the most common and severe form of the disease, after genetic testing confirmed mutations in the SMN1 gene. The parents had already lost a child to the same disorder.

At 32 weeks of pregnancy, doctors administered risdiplam, an FDA-approved SMA drug, to the mother—a first-of-its-kind intervention. The mother took the medication for six weeks, and the baby received an oral dose a week after birth.

Since birth, the child has shown normal muscle development and higher SMN protein levels compared to typical SMA type 1 cases. Nearly three years later, she remains symptom-free, though lifelong monitoring and medication may be required.

This medical milestone offers new hope for early intervention in genetic disorders, potentially changing the future of prenatal treatment.