The goal of this clinical trial is to evaluate the efficacy and safety of the investigational drug ASO-GNAO1 (Tianasen) in pediatric patients with c.607G\>A mutation in the GNAO1 gene associated with epilepsy and neurodevelopmental disorder. The main questions it aims to answer are:
1. Does intrathecal administration of ASO-GNAO1 slow or halt the progression of motor and cognitive symptoms?
2. Is ASO-GNAO1 safe and well-tolerated in this patient population?
3. What is the appropriate therapeutic dose?
This is an open-label study without a placebo control group due to the rare and severe nature of the disease. All participants will receive the active drug.
Participants will:
Receive escalating doses of ASO-GNAO1 via intrathecal injection over a 12-month period.
Undergo frequent neurological assessments, biomarker testing, and safety monitoring.