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Infant Thrives After Groundbreaking In Utero SMA Treatment

3/8/2025

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Summary

Story summary

In a groundbreaking medical achievement, a fetus diagnosed with type 1 spinal muscular atrophy (SMA) received in utero treatment with Risdiplam, a drug that boosts survival motor neuron protein levels. Now, 30 months later, the infant is thriving and symptom-free, igniting hope for enhanced treatments and brighter futures for children battling this condition.

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Story summary

In a groundbreaking medical achievement, a fetus diagnosed with type 1 spinal muscular atrophy (SMA) received in utero treatment with Risdiplam, a drug that boosts survival motor neuron protein levels. Now, 30 months later, the infant is thriving and symptom-free, igniting hope for enhanced treatments and brighter futures for children battling this condition.