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Neurogene ( (NGNE) ) just unveiled an update.
Neurogene Inc. has reported promising interim results from its Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome, highlighting improvements in key areas like motor skills and communication among the first four low-dose pediatric participants. The therapy, which has shown a favorable safety profile, marks significant progress as it addresses the core symptoms of this challenging neurodevelopmental disorder. The company plans to continue the trial with an adolescent/adult cohort and anticipates further updates in 2025, potentially paving the way for a commercial launch.
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