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Epilepsy, Rare Diseases

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

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Published Online: Jan 12th 2022

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

  1. Could you give us a brief overview of tuberous sclerosis complex (TSC), its pathogenesis, clinical manifestations and unmet needs in its treatment? (0:20)
  2. What is ganaxolone and what is the rationale for its use in patients with TSC? (1:11)
  3. What were the aims and design of the clinical study you are presenting? (1:45)
  4. What were the efficacy, safety and tolerability findings of the study? (2:40)
  5. 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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