Clinical Research Directory

Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.

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.

Parkinsonism-Related Oscillations in the Cortico-Basal Ganglia-Thalamic Network During Movement: Beyond the Frequency Range

Expression of hypokinetic and hyperkinetic motor symptoms in Parkinson's disease (PD) is associated with pathological synchronous oscillations of neuronal activity (local field potential/LFP) in the cortico-subcortical network with a wide frequency range. In the present project, we propose to study cortico-subcortical oscillations and their synchronization in patients operated for PD (subthalamic deep brain stimulation (STN-DBS)) during distinct pharmacological and stimulation conditions (hypokinetic and hyperkinetic), using a simple motor task.