touchNEUROLOGY met with Professor Mary Koenig (McGovern Medical School, UTHealth, Houston, TX, USA) to discuss the efficacy and safety findings from the phase 2 clinical study investigating oral ganaxolone for the treatment of tuberous sclerosis complex (NCT04285346).
The abstract ‘Phase 2 Open-label Clinical Study Evaluating Oral Ganaxolone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex‘ (Abstract number: 2.412) was presented at the American Epilepsy Society (AES) Annual Meeting, 3-7 December 2021.
Questions
- Could you give us a brief overview of tuberous sclerosis complex (TSC), its pathogenesis, clinical manifestations and unmet needs in its treatment? (0:20)
- What is ganaxolone and what is the rationale for its use in patients with TSC? (1:11)
- What were the aims and design of the clinical study you are presenting? (1:45)
- What were the efficacy, safety and tolerability findings of the study? (2:40)
- What will be the next step in the clinical development of ganaxolone in this indication? (5:01)
Disclosures: Mary Koenig receives research support from Marinus Pharmaceuticals.
Support: Interview and filming supported by Touch Medical Media.
Filmed as a highlight of AES, 2021
