NGN-401 gene therapy for Rett syndrome showed rapid, durable, and multi-domain developmental gains in all phase I/II participants, with 80% meeting the primary endpoint at 12 months and no milestones lost up to 30 months. The therapy was well tolerated, and robust long-term data support its potential for broad clinical impact.
Based on
This is an AI-generated summary and may contain inaccuracies. Please verify any important information with the original source.