Mary Koenig, AES 2021: Ganaxolone for the Treatment of Tuberous Sclerosis Complex
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.
- 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
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