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Risdiplam Approved for Newborns: Promising SMA Trial Results

8/15/2025

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Summary

Story summary

A clinical trial confirms risdiplam is safe and effective for newborns with spinal muscular atrophy (SMA) from 16 days old.
The study included 23 patients, showing significant motor development; most were walking by age 2.
The FDA has expanded risdiplam's approval to younger patients, focusing on preserving motor function from birth.

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Story summary

A clinical trial confirms risdiplam is safe and effective for newborns with spinal muscular atrophy (SMA) from 16 days old.
The study included 23 patients, showing significant motor development; most were walking by age 2.
The FDA has expanded risdiplam's approval to younger patients, focusing on preserving motor function from birth.