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Groundbreaking Prenatal Drug Defeats Spinal Muscular Atrophy

2/20/2025

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Summary

Story summary

A remarkable two-and-a-half-year-old girl is thriving without any signs of spinal muscular atrophy (SMA), thanks to the innovative drug risdiplam administered before birth. This pioneering approach from St. Jude Children’s Research Hospital opens new doors for prenatal treatments, offering hope for families facing this challenging genetic disorder as she continues her journey of care.

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Story summary

A remarkable two-and-a-half-year-old girl is thriving without any signs of spinal muscular atrophy (SMA), thanks to the innovative drug risdiplam administered before birth. This pioneering approach from St. Jude Children’s Research Hospital opens new doors for prenatal treatments, offering hope for families facing this challenging genetic disorder as she continues her journey of care.