- Lectures / Webinars
- New Developments in the Treatment of TSC-related Epilepsy and TSC-Associated Neuropsychiatric Disorders
New Developments in the Treatment of TSC-related Epilepsy and TSC-Associated Neuropsychiatric Disorders
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ICNC2024
Symposia: Managing The Complexities of TSC - From mTOR Inhibition and CBD To Technology
New Developments in the Treatment of TSC-related Epilepsy and TSC-Associated Neuropsychiatric Disorders
Darcy Krueger
More than 80% of individuals with tuberous sclerosis complex (TSC) will develop epilepsy during childhood. The majority will experience the first seizure before their first birthday and go on to develop medically refractory epilepsy. Closely correlated with uncontrolled epilepsy is the high risk of TSC-Associated Neuropsychiatric Disorders (TAND).
Over the past decade, collaborative teams of basic and translational scientists, clinicians, pharmaceutical and government partners around the world have worked to better understand the underlying pathogenic processes responsible for TSC-related epilepsy and TAND.
In this symposium, we will review recent insights into the underlying neuronal networks disrupted by tubers, the hallmark pathogenic brain lesion of TSC, and how these disruptions are associated with the development of seizures and TAND. We will then focus on the implications of these findings in understanding the results of recently completed landmark epilepsy and TAND prevention clinical trials with the antiseizure medication vigabatrin, EPISTOP (NCT02098759) and PREVeNT (NCT02849457). We will then focus on current clinical trials using sirolimus to target the mTOR pathway directly, STOP2 (NCT04595513), STOP2B:TSC-STEPS (NCT05104983), ViRap (NCT04987463) and RaRE-TS (NCT05534672), as well as provide updates to other ongoing studies and clinical trials in TSC with cannabidiol, ganaxalone, basimglurant, JASPERTM, Regulating TogetherTM, and epilepsy surgery.
Other Lectures in this symposium
International consensus recommendations for the identification and treatment of TSC-Associated Neuropsychiatric Disorders (TAND)
Empowering Families Through Technology: a Mobile-Health Project to Reduce the TAND Identification and Treatment Gap (TANDem)
Improving healthcare for Tuberous Sclerosis Complex (TSC): updated international consensus recommendations for diagnosis, surveillance and management of TSC and real-world implementation