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.
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Change in monthly seizure frequency
Timeframe: Baseline to Week 50
Change in total monthly duration of seizures
Timeframe: Baseline to Week 50
Change in frequency of non-epileptic hyperkinetic and dystonic episodes
Timeframe: Baseline to Week 50
Change in duration of non-epileptic hyperkinetic and dystonic episodes
Timeframe: Baseline to Week 50